ARTICLE | Clinical News

Amicus eyeing 'fastest path' for Pompe's therapy

October 4, 2017 8:23 PM UTC

Amicus Therapeutics Inc. (NASDAQ:FOLD) rose $1.37 to $16.24 on Wednesday after reporting additional efficacy data from a Phase I/II trial showing that combo therapy ATB200/AT2221 improved muscle strength and motor function in patients with Pompe's disease. Amicus CEO John Crowley told BioCentury the company expects to begin a Phase III trial sometime next year, but that Amicus is also working with regulators to "find the fastest path available to bring this medicine to patients."

The trial enrolled 20 patients, including some who had previously received enzyme replacement therapy (ERT), across 3 cohorts: ambulatory ERT-switch patients; non-ambulatory ERT-switch patients; and ERT-naïve patients...

BCIQ Company Profiles

Amicus Therapeutics Inc.

BCIQ Target Profiles

Acid alpha glucosidase (GAA)