Scientific Findings Reveal that Cellular Damage Alters Trafficking for Key Proteins Involved in Muscle Membrane Integrity and Muscle Repair
Preclinical Studies Demonstrate Reversal of Cellular Damage and Significant Improvements in Muscle Strength in GAA Knock-out Mice After Treatment with Amicus Novel Pompe Treatment Paradigm
Additional Phase 1/2 Clinical Data to be Presented at Wednesday Afternoon Poster Session (
In previously reported preclinical studies, ATB200 was associated with increased tissue enzyme levels and reduced glycogen levels in muscle, which was further improved when AT2221 was co-administered with ATB200. For the first time at WORLDSymposium, Hung Do, PhD, Chief Science Officer of
Key Scientific Findings
Key Highlights on New Preclinical Data for ATB200/AT2221 in GAA Knock-Out Mice
Dr. Do stated, "Our latest preclinical studies give us further confidence that ATB200/AT2221 has been optimally designed for efficient targeting to muscles. The results build upon our prior preclinical studies showing that ATB200/AT2221 not only clears accumulated glycogen but importantly, significantly reversed muscle damage and increased muscle function. These studies have also been invaluable for gaining important insights into the role of protein mistrafficking as contributing factors towards muscle weakness and disrepair in Pompe disease, and the potential for ATB200/AT2221 to reverse cellular dysfunction to restore muscle structure and increase muscle strength."
ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose 6-phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone. In preclinical studies, ATB200 was associated with increased tissue enzyme levels and reduced glycogen levels in muscle, which was further improved when AT2221 was co-administered with ATB200.
About Pompe Disease
Pompe disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscles and other tissues. Progressive accumulation of glycogen is believed to lead to the morbidity and mortality associated with Pompe disease, including muscle weakness and respiratory insufficiency.
This press release contains "forward- looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to encouraging pre-clinical data from a study to investigate ATB200/AT2221 in a knock-out mouse model and the potential implications on these data for the future advancement and development of ATB200/AT2221 in humans. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "confidence," "encouraged," "potential," "plan," "targets," "likely," "may," "will," "would," "should" and "could," and similar expressions or words identify forward-looking statements. The forward looking statements included in this press release are based on management's current expectations and belief's which are subject to a number of risks, uncertainties and factors, including that the pre-clinical data will not be predictive of future results in humans, and that preliminary human clinical data based on a small patient sample and reported before completion of the study will not be predictive of future results, that results of additional preliminary data or data from the completed study or any future study will not yield results that are consistent with the pre-clinical or preliminary data presented, that the Company will be not able to demonstrate the safety and efficacy of ATB200/AT2221, that later study results will not support further development, or even if such later results are favorable, that the Company will not be able to successfully complete the development of, obtain regulatory approval for, or successfully commercialize ATB200/AT2221. In addition, all forward looking statements are subject to the other risks and uncertainties detailed in our Annual Report on Form 10-K for the year ended
Amicus Therapeutics Sara PellegrinoSenior Director, Investor Relations firstname.lastname@example.org (609) 662-5044 Media: MWW PR Sid Dinsaysdinsay@mww.com (646) 381-9017
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