Amicus presents four-year data from PROPEL study of Pombiliti, Opfolda

Amicus (FOLD) Therapeutics announced the presentation of new 4-year muscle function, muscle strength and biomarker endpoints from the PROPEL open-label extension study of cipaglucosidase alfa-atga + miglustat in adults with late-onset Pompe disease at the International Congress of Inborn Errors of Metabolism in Kyoto, Japan. The new analysis, “208-week efficacy and safety of cipaglucosidase alfa plus miglustat in patients with late-onset Pompe disease treated from PROPEL baseline: muscle function and biomarkers,” was conducted on the 82 patients who were randomized to cipa+mig in PROPEL and who enrolled in the open label extension. Pombiliti + Opfolda is indicated in the U.S. for the treatment of adult patients with LOPD weighing greater than or equal to40 kg and who are not improving on their current ERT. In the U.S., Pombiliti + Opfolda is not indicated for patients naive to ERT treatment. The data summarized below are from the ERT-experienced group in the OLE. Pulmonary function data from the PROPEL OLE will be presented separately at an upcoming conference. The mean age was 48.8 years and the median duration of prior ERT in the ERT-experienced cohort at PROPEL baseline was 7.5 years. In the ERT-experienced group, the measures of muscle function, muscle strength and biomarkers improved and/or were sustained out to 208 weeks. Mean change from PROPEL baseline to week 208 were as follows: Percent predicted six-minute walk distance, +2.3%; Lower extremity manual muscle test score, +1.6 points; Gait-Stairs-Gowers-Chair score, +0.1 points; PROMIS physical function score, +1.2 points; Serum creatine kinase levels, -160.0 serum CK; and, Urine hexose tetrasaccharide, -1.9 mmol/mol creatinine. No new safety signals were identified. Up to week 208, 41 patients experienced treatment-related treatment-emergent adverse events, leading to four cipa+mig discontinuations, and two patients experienced serious treatment-related TEAEs, leading to two cipa+mig discontinuations. One patient experienced a TEAE unrelated to study treatment, which led to death; the patient had discontinued from the study prior to death.