Newly Released Data from Phase 1/2 Shows Promise for Amicus' Pompe Drug

Mathew Shanley

This morning, Amicus Therapeutics announced positive results from a Phase 1/2 study of the company’s ATB200/AT2221 in patients with Pompe disease.

The latest clinical data were featured in a late-breaker poster at the 22nd International Congress of the World Muscle Society, and the information conveyed remained consistent with previous outcomes. The therapy improved the 6-minute walk test (6MWT) distance and other measures of motor function, stability or increases in forced vital capabity (FVC), among its other promising effects.

A deficiency in acid alpha-glucosidase (GAA) causes the rare inherited lysosomal storage disorder Pompe disease. ATB200/AT2221 is a fixed-dose combination therapy that consists of a recombinant human GAA (rhGAA) enzyme with an optimized carbohydrate structure (ATB200), administered with a small molecule pharmacological chaperone (AT2221).

"These remarkable data from our Pompe clinical study of ATB200/AT2221 have once again exceeded our expectations. 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. Taken together the strength of these results suggest the effect of ATB200/AT2221 may be very clinically meaningful for people living with Pompe disease,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics in a press release.

To date, adverse events (AEs) in clinical trial have been mostly mild and temporary. Infusion-associated reactions (IARs) has been minimal. While the study was focused on safety and muscle strength outcomes, the study did find that treatment with ATB200/AT2221 resulted in reductions in key disease biomarkers across all patient cohorts, suggesting a positive effect on muscle cells.

The hopes to begin a Phase 3 study in early 2018. Crowley stated that company executives are expected to take early-stage data to regulators in a pursuit to accelerate approval.

Mark Roberts, MD, the principal investigator in the study added: “I believe that the results from this Phase 1/2 clinical study show striking improvements in functional measures and key biomarkers during the first six months of treatment, in addition to continued, further benefit out to nine months. I am especially intrigued by the six-minute walk distance and other motor function tests in the ERT-switch patients who historically have declining motor function following two or more years of treatment. These clinical data are compelling and suggest that ATB200/AT2221 has the potential to shift the treatment paradigm for Pompe disease.”

Shares in Amicus’ stock jumped 21% today because of the positive data, and while it’s early in the assessment of the drug, Amicus is confident that the improved strength of subjects in the study means they’re on to something big.

“We are committed to working collaboratively with regulators to determine the fastest regulatory pathways that may be available to bring this new treatment paradigm to as many patients living with Pompe disease globally, as quickly as possible," Crowley said.

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