- Amicus Therapeutics (NASDAQ:FOLD) announces additional positive data from its Phase 1/2 clinical trial assessing ATB200/AT2221 in patients with Pompe disease, an inherited lysosomal storage disorder. The results were presented at the World Muscle Society Conference in France.
- After six months of treatment, muscle function improved in 88.9% (n=16/18) of patients and was stable in the other two. Six-minute walk test distance improved at month 6 and was durable to month 9.
- Pulmonary function either improved or was stable at months 6 and 9.
- ATB200/AT2221 is a new treatment paradigm consisting of ATB200, a recombinant human enzyme called acid alpha-glucosidase with enhanced uptake characteristics, and AT2221, a pharmacological chaperone that improves activity and stability.
- Management will host a conference call this morning at 8:30 am ET to discuss the data.
- Shares are up 16% premarket.
- Previously: Amicus' ATB200/AT2221 shows treatment effect in early-stage Pompe study; shares ahead 7% premarket (May 15)
- Now read: Move Over Yellen, It's Tax Cut Time!
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