Amicus Releases More Positive Data at WORLDSymposium

Today at the 14^th Annual WORLDSymposium, Amicus Therapeutics presented a late-breaker poster and corresponding oral presentation, announcing additional positive results from the company’s Phase 1/2 clinical study of ATB200/AT2221 in patients with Pompe disease.

In the global study, patients treated with ATB200/AT2221 for up to 12 months displayed improvements in 6-minute walk test (6MWT) distance and other measures of motor function, stability, or increases in forced vital capacity (FVC), and durable reductions in biomarkers of muscle damage and disease substrate.

At the 22^nd International Congress of the World Muscle Society in October, Amicus presented its initial round of positive data from the study, exhibiting the therapy’s ability to remain consistent with previous outcomes.

Pompe disease, a genetic lysosomal storage disorder, is caused by a deficiency in acid alpha-glucosidase (GAA). 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).

“We continue to be impressed by the clinical data for our novel Pompe treatment paradigm ATB200/AT2221,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics stated. “These latest data, in more patients and over longer periods, have continued to show substantial improvements in functional outcomes in nearly all patients, which are aligned with the persistent and durable reductions in key biomarkers of muscle damage and disease substrate. On the heels of these data for ATB200/AT2221 we look forward to continuing our discussions with global regulators to define the best and fastest pathway to deliver this critically important medicine to people living with Pompe disease.”

Primary objectives of the open-label study are to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ATB200/AT2221 over an 18-week primary treatment period followed by a long-term extension. In the study, 20 patients were enrolled, of which one dropped out due to travel burden, leaving available data on functional outcomes from 19 participants. Muscle function improved in 16 of 19 patients at month 9, and improved in 10 out of 10 patients with available data at month 12.

Motor function, defined by the 6MWT, improved consistently in both enzyme replacement therapy (ERT)-naïve and ERT-switch patients with continued clinical benefit observed through month 12. Muscle function and strength was improved in the upper extremities of 3 of the 4 non-ambulatory ERT-switch patients from baseline to month 9. Pulmonary function improved in ERT-naïve patients and was generally stable in ERT-switch patients.

In the video below, Crowley sits down with Rare Disease Report for an exclusive interview about the data and his company's presentation:

Crowley: We were really privileged today to be able to present additional positive data on our Phase 1 / 2 study with our new medicine for Pompe Disease; an investigational medicine where we are looking for the ability of this enzyme to target muscle cells and reduce glycogen and, hopefully, to lead to improvements on patients in a number of measures. We had previously provided data back in October, showing a successful Phase 1/2 study - both in people who have taken enzyme replacement therapy before in an improved product and then switched to our new investigational drug and, then also for people who have never received enzyme replacement therapy before. It's very exciting. There, we were able to show that people were able to walk further. In some cases, some people, in some measures, were able to breathe better; that was the goal. Can we have an impact on patients’ lives? We were very pleased to show that.

Now, at this conference, we were able to show data on even more patients, continuing those positive trends. We were also able to show that it goes out in time. Back in the Fall, we showed the data at six months. Now we have the data in 9 months and in some cases 12 months. Out to a year, showing the improvements in patients. So, we are really pleased with this. We think it shows the magnitude of response, it shows consistency which is so important to cross patients and also the durability across time. So very, very positive results for us and certainly a very important program for Amicus.

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