Full Press Release Details
PHILADELPHIA, March 15, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced one oral presentation and two posters highlighting its development program for Pompe disease will be included at the 2021 MDA Virtual Clinical & Scientific Conference to be held March 15-18, 2021.
Oral Platform Presentation: Thursday, March 18, 5:30 – 5:45 p.m. ET
Efficacy and safety of AT-GAA (cipaglucosidase alfa/miglustat) versus alglucosidase alfa/placebo in late-onset Pompe disease (LOPD): A phase 3 trial (PROPEL) Presenter: Tahseen Mozaffar, MD, FAAN, University of California, Irvine, CA, USA
Poster Presentations:
Characterization of Response to Enzyme Replacement Therapy in Patients With Late-Onset Pompe Disease: A Retrospective Chart Review Presenter: Priya Kishnani, MD, MBBS, Duke University Medical Center, Durham, NC, USA
Enhancing Delivery of Acid Alpha-Glucosidase (GAA) to Skeletal Muscle in Pompe Disease (PD): Key Challenges and Attributes of AT-GAA Presenter: Nithya Selvan, Ph.D., Amicus Therapeutics, Philadelphia, PA, USA
The posters and presentation will be made available on the Amicus website following their respective presentations at the conference.
For more information on the 2021 MDA Virtual Clinical & Scientific Conference, please visit www.mdaconference.org.
AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa (ATB200), a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly bis-phosphorylated mannose-6 phosphate (bis-M6P) glycans, to enhance uptake into cells, administered in conjunction with miglustat (AT2221), a stabilizer of cipaglucosidase alfa. In preclinical studies, AT-GAA was associated with increased levels of the mature lysosomal form of GAA and reduced glycogen levels in muscle, alleviation of the autophagic defect and improvements in muscle strength.
In addition, Amicus is enrolling an open-label, uncontrolled, multicenter study to evaluate the PK, safety, efficacy, and PD of AT-GAA in pediatric patients aged 12 to <18 years with LOPD (ATB200-04). More information, including a list of participating sites, is available at www.clinicaltrials.gov: NCT03911505
Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA levels lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. The disease can be debilitating and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.
About Amicus Therapeutics
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