Amicus Therapeutics, Inc.(NASDAQ:FOLD) has announced positive initial results from a global Phase 1/2 research to investigate ATB200/AT2221.Amicus Therapeutics is a biotechnology company, and a top Biotech Stocks that mainly focuses on rare and orphan diseases globally.
ATB200/AT2221 is a novel model of treatment that is made up of ATB200 which is a peculiar human acid alpha-glucosidase (rhGAA) enzyme. The acid has a carbohydrate structure which is optimized to increase the rate of uptake. It is administered together with AT2221a pharmacological chaperone.
During the presentation of the results at the WORLD Symposium, the company’s Chairman and Chief Executive Officer John F. Crowley said they are still in the process of cascading all the information from the study in Pompe Disease patients. “We are very pleased to share these additional positive preliminary clinical results here at the WORLD Symposium as we continue with the cascade of data from this important study in Pompe Disease patients,” he said. He adds that apart from high level safety measures employed in the study, they have evidence to allude that the Amicus treatment model is reducing some important elements of the disease in both the first ERT-treatment naïve patients as well as ERT-switch patients.
Crowley adds that using this information, they are able to reduce on these biomarkers which he acknowledges is very encouraging. Crowley says to this date, no other publication for other Pompe ERTs has shown such levels of reduction in biomarkers. He adds that by the fact that the biomarkers have showed a constant reduction in a big number of patients during the first 18 weeks is an indication that the effects of the study may be durable.
ATB200 is characterized with increase in levels of tissue enzyme and a reduction in levels of glycogen in muscles. Amicus Therapeutics is carrying out Phase 1/2 study to establish the safety, level of tolerance, pharmacodynamics and pharmacokinetics (PK) of ATB200/AT2221. Pompe disease is a lysosomal storage disease which comes as a result of lack of acid alpha-glucosidase (GAA), which lead to increase in levels of glycogen in muscles and body tissues. High levels of glycogen are believed to cause mortality and morbidity associated with Pompe disease.