Amicus Reports Additional Positive Results in Pompe Trial Combination Therapy

October 5, 2017

Rare Daily Staff

Amicus Therapeutics announced additional positive results from a global phase 1/2 clinical study to investigate ATB200/AT2221, its combination therapy for patients with Pompe disease, an inherited lysosomal storage disorder that causes progressive muscle deterioration.

ATB200/AT2221 is a novel treatment that consists of ATB200, a unique recombinant human acid alpha-glucosidase enzyme with optimized carbohydrate structures to enhance uptake, co-administered with AT2221, a pharmacological chaperone that stabilizes the enzyme within the body.

Consistent with previous results, patients who completed six months of treatment with ATB200/AT2221 showed improvements in six-minute walk test distance and other measures of motor function, stability or increases in forced vital capacity, and further reductions in biomarkers of muscle damage and disease substrate. The findings were consistent with results reported in initial patients who completed nine months of treatment.

The clinical results were featured at the 22nd International Congress of the World Muscle Society in a late-breaker poster. Amicus said it plans to continue a series of collaborative discussions with regulators in the United States and European Union, and expects to provide an update in the first half of 2018.

“The consistency, durability, and magnitude of the functional outcomes align with significant and continued reductions in key biomarkers of muscle damage and disease substrate, across patients, across cohorts, and over significant periods of time,” said John Crowley, chairman and CEO of Amicus. “These remarkable data from our Pompe clinical study of ATB200/AT2221 have once again exceeded our expectations.”

Safety and tolerability data in all 20 patients reflect a maximum of 72 weeks of treatment. To date, adverse events have been generally mild and transient. ATB200/AT2221 has resulted in a low rate of infusion-associated reactions following more than 400 infusions (three events in two patients).

October 5, 2017

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