Amicus down 5% on Pompe study questions

Amicus Therapeutics (FOLD -5.2%) slips on below-average volume in apparent response to an analyst’s question during its Q2 conference call this morning about its late-stage study, PROPEL, evaluating the combination of of ATB200 and AT2221 in patients with late-onset Pompe disease (LOPD). The BofA analyst questioned whether the primary endpoint of the 6-minute walk test (6MWT) is better than Sanofi’s lung function primary endpoint in its Phase 3 LOPD study, COMET, of enzyme replacement therapy avalglucosidase alfa (neoGAA), adding that Sanofi may announce preliminary data sooner.

Pompe disease is a rare inherited disorder in which the sugar molecule glycogen builds up in cells due to the absence or dysfunction in an enzyme called glucosidase. Symptoms include weak muscles, enlarged liver and breathing difficulties. It is typically diagnosed in infants, but late-onset Pompe can occur in adults who have less severe Pompe (more glucosidase activity) hence the question about the 6MWT since symptoms develop slowly over time. Respiratory difficulties are frequently the first signs of the disease.

https://seekingalpha.com/news/3489643-amicus-5-percent-pompe-study-questions