Amicus Therapeutics will present new findings on Fabry and Pompe diseases at the 21st Annual WORLDSymposium™ 2025.
Quiver AI Summary
Amicus Therapeutics announced that it will showcase two oral presentations and 20 posters related to its research on Fabry and Pompe diseases at the 21st Annual WORLDSymposium™ 2025 taking place from February 3-7 in San Diego, CA. CEO Bradley Campbell emphasized the company's commitment to advancing treatments for patients with these rare diseases. The presentations will feature significant studies, including a first-in-class enzyme stabilizer for late-onset Pompe disease and improvements in walk distance and lung function among patients switching treatments. The event is expected to draw various stakeholders interested in the latest research and clinical advancements in lysosomal diseases. Amicus is focused on developing innovative medicines for rare conditions, integrating extensive patient feedback and data into its research efforts.
Potential Positives
- Amicus Therapeutics is demonstrating scientific leadership in the treatment of Fabry and Pompe diseases through presentations at the prestigious WORLDSymposium 2025.
- The company will showcase significant research, including two oral presentations highlighting important clinical improvements for patients switching treatment regimens in Pompe disease.
- Presence at WORLDSymposium with 20 posters indicates a strong commitment to advancing research and development in the field of rare diseases, enhancing the company's visibility and credibility.
- Amicus reinforces its dedication to patient-focused outcomes as part of their mission to deliver transformative medicines for rare diseases, which may strengthen stakeholder trust and support.
Potential Negatives
- Despite presenting at a significant symposium, the company may be facing scrutiny regarding the efficacy and safety of their drug candidates, particularly given the switch from alglucosidase alfa to cipaglucosidase alfa plus miglustat in the PROPEL study.
- The focus on rare diseases and the company's ongoing development efforts may highlight the challenges and limitations in their current pipeline, raising concerns about the accessibility and practical benefits of their treatments for patients.
- The press release's heavy reliance on presentations and posters may indicate a lack of substantial new trial results or drug approvals, which could affect investor confidence and market perception.
FAQ
What is the main focus of Amicus Therapeutics?
Amicus Therapeutics focuses on discovering and delivering innovative medicines for patients with rare diseases, particularly Fabry and Pompe diseases.
When is WORLDSymposium 2025 being held?
WORLDSymposium 2025 will take place from February 3-7, 2025, in San Diego, CA.
How many presentations will Amicus Therapeutics showcase at WORLDSymposium 2025?
Amicus Therapeutics will present two oral presentations and 20 posters at WORLDSymposium 2025.
Who are the presenters for the oral presentations at WORLDSymposium?
The presenters include Robert J. Hopkin and Benedikt Schoser, focusing on Pompe disease and its management.
What topics will be covered in the poster sessions?
Poster sessions will cover various aspects of Fabry and Pompe diseases, including patient experiences, treatment outcomes, and safety.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
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Full Release
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 (P oster #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 (P oster #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 WORLD
Symposium
WORLD
Symposium
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
[email protected]
(609) 662-3809
Media:
Amicus Therapeutics
Diana Moore
Vice President, Corporate Communications
[email protected]
(609) 662-5079
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