Benzinga | Feb 11, 2021 04:04PM EST

Amicus Reports AT-GAA Shows Clinically Meaningful & Significant Improvements in Both Musculoskeletal and Respiratory Measures in Late-Onset Pompe Disease Compared to Standard of Care in Pivotal Phase 3 PROPEL Study

Rolling BLA submission for AT-GAA planned for completion in Q2 and other global regulatory submissions for approval expected throughout 2021

Patients switching to AT-GAA from the approved standard of care ERT (alglucosidase alfa) walked on average 17 meters farther (p=0.046)

Patients switching to AT-GAA also showed an improvement in percent-predicted forced vital capacity (FVC), the most important measure of respiratory function in Pompe disease, compared to a decline in patients treated with alglucosidase alfa (FVC Diff. 4.1%; p=0.006)

AT-GAA showed a nominally statistically significant and clinically meaningful difference for superiority on the first key secondary endpoint of FVC compared to patients treated with alglucosidase alfa (FVC Diff. 3.0%; p=0.023)

In the combined study population of ERT switch and ERT na?ve patients, AT-GAA outperformed alglucosidase alfa by 14 meters (21m compared to 7m) on the primary endpoint and was not statistically significant for superiority (p=0.072)

Improvements in the two important biomarkers of Pompe disease (Hex-4 and CK) for the combined study population significantly favored AT-GAA compared to alglucosidase alfa (p<0.001)

Conference Call at 4:30pm EST today with results to be presented in a platform presentation session at the 17th Annual WORLDSymposium(tm) 2021 on Friday, February 12 at 9:30am