Amicus Therapeutics Announces Presentations and Posters at the 21st Annual WORLDSymposium™ 2025

Rhea-AI Summary

Amicus Therapeutics (FOLD) announced its participation at the 21st Annual WORLDSymposium™ 2025, featuring two oral presentations and 20 posters focused on Fabry and Pompe diseases. The presentations include key findings on miglustat, a first-in-class enzyme stabilizer for late-onset Pompe disease, and improvements in patients switching to cipaglucosidase alfa plus miglustat treatment.

The extensive poster sessions cover various aspects of both diseases, including patient experiences, treatment outcomes, and clinical studies. Notable presentations include long-term safety data of migalastat in Fabry disease patients, real-world evidence studies, and results from the PROPEL study showing clinical improvements in Pompe disease patients.

Positive

• Significant clinical improvements demonstrated in PROPEL study for Pompe disease patients
• Long-term safety and efficacy data available for migalastat in adolescent Fabry patients
• Comprehensive research portfolio with 22 total presentations showing strong R&D pipeline

Negative

• None.

PRINCETON, N.J., Feb. 03, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced that two oral presentations and 20 posters across its development programs will be included at the 21st Annual WORLDSymposium™ 2025, being held February 3-7, 2025 in San Diego, CA.

“As part of our ongoing efforts to advance our scientific leadership in Fabry and Pompe diseases, Amicus researchers will present two oral presentations and 20 posters at this year’s WORLDSymposium,” said Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc. “We’re proud of the progress we are making for patients living with Fabry and Pompe and are committed to build on this momentum in pursuit of our mission to develop and deliver transformative medicines for people living with rare diseases.”

Oral Platform Presentation:

Pompe Disease:

Abstract Title: Miglustat: A first-in-class enzyme stabilizer for late-onset Pompe disease

• Presenter: Robert J. Hopkin, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, U.S.A.
• Date and time: Wednesday, February 5, 11:00 a.m. PST

Abstract Title: Clinically important improvements in 6-minute walk distance (6MWD) and forced vital capacity (FVC) in adults with late-onset Pompe disease (LOPD) switching from alglucosidase alfa (alg) to cipaglucosidase alfa plus miglustat (cipa+mig) in the PROPEL study

• Presenter: Benedikt Schoser, Ludwig-Maximilians-University, Munich, Germany
• Date and time: Thursday, February 6, 1:30 p.m. PST

Poster Sessions:

Fabry Disease:

Abstract Title: Exploring the lived experiences and care perceptions of females living with Fabry in the US, Canada, and Japan (Poster #8)

• Presenter: Julia Alton, Canadian Fabry Association, Ontario, Canada
• Date and time: Tuesday, February 4, 3:30 – 5:30 p.m. PST
• Location: Kiosk 2-B
  

Abstract Title: Exploring the demographics of the diagnosed Fabry population in Canada (Poster #9)

• Presenter: Julia Alton, Canadian Fabry Association, Ontario, Canada
• Date and time: Tuesday, February 4, 3:30 – 5:30 p.m. PST
• Location: Kisok 3-A
  

Abstract Title: Stable: A protein engineering platform for enhancing stability and delivery of alpha-galactosidase A for Fabry disease therapy (Poster #358)

• Presenter: Tobias Willer, Amicus Therapeutics, Philadelphia, PA, U.S.A.
• Date and time: Wednesday, February 5, 3:30 – 5:30 p.m. PST
• Location: Kiosk 31-A
  

Abstract Title: Long-term safety and efficacy of migalastat in adolescent patients with Fabry disease: Results from the ASPIRE study and open-label extension (Poster #283)

• Presenter: Uma Ramaswami, Royal Free London NHS Foundation Trust and Genetics and Genomic Medicine, London, U.K.
• Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
• Location: Kiosk 32-D
  

Abstract Title: Treatment satisfaction in patients with Fabry disease: Patient-reported outcomes from the FollowME Fabry Pathfinders registry and the SATIS-Fab study (Poster #90)

• Presenter: Ulla Feldt-Rasmussen, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark
• Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
• Location: Kiosk 1-C
  

Abstract Title: FollowME Fabry Pathfinders Registry: Patient-reported outcomes in a cohort of patients on migalastat treatment for median 4 years (Poster #91)

• Presenter: Ulla Feldt-Rasmussen, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark
• Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
• Location: Kiosk 1-D
  

Abstract Title: Quality of life of migalastat-treated adolescents with Fabry disease: Results from the ASPIRE study and open-label extension (Poster #142)

• Presenter: Robert J. Hopkin, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, U.S.A.
• Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
• Location: 15-C
  

Abstract Title: Trial in progress: An open-label study (AT1001-025 RENEW) to evaluate the safety and pharmacokinetics of migalastat in patients with Fabry disease and amenable GLA variants with severe renal impairment or end-stage renal disease treated with dialysis (Poster #136)

• Presenter: Jennifer Hiros, Amicus Therapeutics, Princeton, NJ, U.S.A.
• Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
• Location: Kisok 12-D
  

Abstract Title: An observational cohort study evaluating outcomes in patients with Fabry disease treated with migalastat in a real-world setting in France (MIGA-Fab) (Poster #185)

• Presenter: Didier Lacombe, Bordeaux University Hospital, Bordeaux, France
• Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
• Location: Kiosk 24-C
  

Pompe Disease:

Abstract Title: Non-invasive magnetic resonance imaging monitoring of glycogen accumulation in a mouse model of Pompe disease (Poster #361)

• Presenter: Nirbhay Yadav, F. M. Kirby Research Center for Functional Brain Imaging, Kennedy Krieger Institute, Baltimore, MD, U.S.A.
• Date and time: Tuesday, February 4, 3:30 – 5:30 p.m. PST
• Location: Kiosk 21-D
  

Abstract Title: Miglustat: A first-in-class enzyme stabilizer for late-onset Pompe disease (Poster #139)

• Presenter: Robert J. Hopkin, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, U.S.A.
• Date and time: Wednesday, February 5, 3:30 – 5:30 p.m. PST
• Location: Kiosk 12-A
  

Abstract Title: Reaching consensus on comprehensive and achievable monitoring for adults with late-onset Pompe disease in the UK (Poster #71)

• Presenter: Patrick Deegan, Cambridge University Hospitals NHS Foundation Trust (CUH), Cambridge, U.K.
• Date and time: Wednesday, February 5, 3:30 – 5:30 p.m. PST
• Location: Kiosk 4-D
  

Abstract Title: Pompe disease in Sweden: A real-world evidence study investigating disease burden, treatment patterns for enzyme replacement therapy and concomitant medications (Poster #248)

• Presenter: Sofia Nordin, Amicus Therapeutics, Copenhagen, Denmark
• Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
• Location: Kiosk 26-D
  

Abstract Title: Patient perspective of participation in clinical studies in LOPD: Structured exit interviews from cipaglucosidase alfa plus miglustat studies (Poster #102)

• Presenter: Jamie Gault, Amicus Therapeutics, Princeton, NJ, U.S.A.
• Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
• Location: Kisok 15-B
  

Abstract Title: POM-005: A global, prospective, observational registry of people living with Pompe disease (Poster #225)

• Presenter: Paul McIntosh, University of Pennsylvania, Philadelphia, PA, U.S.A.
• Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
• Location: Kiosk 30-A
  

Abstract Title: Clinically important improvements in 6-minute walk distance (6MWD) and forced vital capacity (FVC) in adults with late-onset Pompe disease (LOPD) switching from alglucosidase alfa (alg) to cipaglucosidase alfa plus miglustat (cipa+mig) in the PROPEL study (Poster #303)

• Presenter: Benedikt Schoser, Ludwig-Maximilians-University, Munich, Germany
• Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
• Location: Kiosk 36-B
  

Abstract Title Outcomes of a pediatric patient with late-onset Pompe disease switching from high-dose, high-frequency alglucosidase alfa to standard-dose cipaglucosidase alfa plus miglustat (Poster #70)

• Presenter: Stephanie DeArmey, Duke University Medical Center, Durham, NC, U.S.A.
• Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
• Location: Kiosk 5-C
  

Abstract Title: Cipaglucosidase alfa plus miglustat in late-onset Pompe disease: two non-ambulatory patients switching from high-dose, high-frequency alglucosidase alfa (Poster #46)

• Presenter: Barry J. Byrne, University of Florida, Gainesville, FL, U.S.A.
• Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
• Location: Kiosk 7-B
  

Abstract Title: Post-baseline outcomes of the UK Early Access to Medicines Scheme registry for cipaglucosidase alfa plus miglustat in late-onset Pompe disease (Poster #288)

• Presenter: Mark Roberts, Salford Royal NHS Foundation Trust, Salford, U.K.
• Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
• Location: Kiosk 33-B
  

Abstract Title: Correction of glycogen accumulation in muscle, heart and CNS in a pre-clinical model of hematopoietic stem cell gene therapy for Pompe disease (Poster #351)

• Presenter: Slawomir Wantuch, UCL Great Ormond Street Institute of Child Health, London, U.K.
• Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
• Location: Kisok 38-D
  

About WORLDSymposium
WORLDSymposium is designed for basic, translational and clinical researchers, patient advocacy groups, clinicians, and all others who are interested in learning more about the latest discoveries related to lysosomal diseases and the clinical investigation of these advances. Each year, WORLDSymposium presents the latest information from basic science, translational research, and clinical trials for lysosomal diseases. For more information, please visit www.worldsymposia.org.

About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company’s website at www.amicusrx.com, and follow on X and LinkedIn.

CONTACTS:

Investors:
Amicus Therapeutics
Andrew Faughnan
Vice President, Investor Relations
afaughnan@amicusrx.com
(609) 662-3809

Media:
Amicus Therapeutics
Diana Moore
Vice President, Corporate Communications
dmoore@amicusrx.com
(609) 662-5079

FOLD–G

FAQ

What are the key findings presented by Amicus Therapeutics (FOLD) at WORLDSymposium 2025?

Amicus presented two oral presentations and 20 posters, highlighting improvements in Pompe disease treatment with cipaglucosidase alfa plus miglustat, and long-term safety data for migalastat in Fabry disease patients.

What clinical improvements were shown in the PROPEL study for FOLD's Pompe disease treatment?

The PROPEL study demonstrated important improvements in 6-minute walk distance (6MWD) and forced vital capacity (FVC) in adults with late-onset Pompe disease who switched to cipaglucosidase alfa plus miglustat treatment.

What are the long-term safety results for FOLD's migalastat in Fabry disease treatment?

The ASPIRE study and open-label extension demonstrated long-term safety and efficacy of migalastat in adolescent patients with Fabry disease, including quality of life improvements.

How many presentations did Amicus Therapeutics (FOLD) have at WORLDSymposium 2025?

Amicus Therapeutics presented a total of 22 scientific updates, consisting of two oral presentations and 20 poster presentations across their Fabry and Pompe disease programs.