Amicus Therapeut Stock Price, News & Analysis

Amicus Therapeutics (Nasdaq: FOLD) will host a conference call and live audio webcast on May 10, 2021, at 8:30 a.m. ET, to discuss its financial results for Q1 2021, ended March 31, 2021. Investors can join by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international), using conference ID: 5767104. A replay will be available for seven days starting at 11:30 a.m. ET. Amicus focuses on developing medicines for rare metabolic diseases, emphasizing a patient-centric approach.

Amicus Therapeutics (Nasdaq: FOLD) announced its participation in the 2021 MDA Virtual Clinical & Scientific Conference from March 15-18, showcasing advancements in its Pompe disease program. The event features an oral presentation on the phase 3 trial of AT-GAA (cipaglucosidase alfa/miglustat) and two poster presentations. The oral presentation will occur on March 18, focusing on efficacy and safety comparisons with existing treatments. Materials will be available on Amicus's website post-presentation.

Amicus Therapeutics reported $260.9 million in total Galafold revenue for 2020, marking a 43% year-on-year increase. The company anticipates 2021 revenue for Galafold to be between $300 million and $315 million, driven by robust global growth. The AT-GAA BLA submission for Pompe disease is scheduled for completion in 2Q21, alongside other global submissions. The FDA granted Fast Track Designation for the CLN6 gene therapy, bolstering its pipeline. As of December 31, 2020, Amicus held $483.3 million in cash, sufficient for self-sustainability.

Amicus Therapeutics (Nasdaq: FOLD) announced that its senior leadership team will present at two upcoming virtual investor conferences. The SVB Leerink 10th Annual Global Healthcare Conference is scheduled for February 25, 2021, at 12:00 p.m. E.T., followed by the Cowen 41st Annual Health Care Conference on March 3, 2021, at 12:20 p.m. E.T. Live audio webcasts of these presentations will be available on Amicus Therapeutics' corporate website.

The company focuses on developing innovative treatments for rare metabolic diseases.

Amicus Therapeutics (Nasdaq: FOLD) will host a conference call on March 1, 2021, at 8:30 a.m. ET to discuss its financial results for the year ended December 31, 2020. Investors can join the call by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international) with conference ID 6959755. A live audio webcast will also be available on the company's website. A replay will be accessible for seven days following the call. Amicus is focused on developing high-quality medicines for rare metabolic diseases.

Amicus Therapeutics (FOLD) announced positive topline results from its Phase 3 PROPEL Trial for AT-GAA, a therapy for late-onset Pompe disease. Patients switching to AT-GAA from alglucosidase alfa walked an average of 17 meters farther (p=0.046) and showed a 4.1% improvement in forced vital capacity (FVC) (p=0.006). AT-GAA exhibited significant improvements in biomarkers Hex-4 and CK (p<0.001). A rolling Biologics License Application (BLA) submission is expected to complete in Q2 2021, while further global regulatory submissions are anticipated throughout the year.

Amicus Therapeutics (Nasdaq: FOLD) announced initial positive results from its Phase 1/2 study of AT-GTX-502, a gene therapy for CLN3 Batten disease, during the 17th Annual WORLD Symposium 2021. Initial safety data from four children indicate treatment was well tolerated with mostly mild adverse events. Importantly, efficacy data show potential disease stabilization, with a mean UBDRS score change of +0.07 versus +2.86 in untreated subjects. Amicus plans to submit an IND for the next clinical study in the second half of 2021.

Amicus Therapeutics (Nasdaq: FOLD) has released promising preclinical data for its AAV gene therapy program targeting Fabry disease. The results, presented at the 17th Annual WORLD Symposium, indicate that the engineered transgene, AT-GTX-701, shows significantly greater substrate reduction in relevant tissues compared to the wildtype transgene. Notably, initial studies demonstrate a dose-dependent response, with the lead engineered transgene achieving near complete substrate reduction at the highest dose. Ongoing studies focus on further development of this gene therapy and its potential for other lysosomal disorders.

Amicus Therapeutics (Nasdaq: FOLD) announced participation in the 17th Annual WORLD Symposium™ 2021 with three oral presentations and ten posters focused on its development programs for Lysosomal Disorders. Notable presentations include findings related to the α-galactosidase-A deficiency in Fabry disease and developments in gene therapy for various lysosomal storage diseases. The symposium aims to foster discussions on research and clinical applicability in lysosomal diseases, promoting advancements in patient care and treatment approaches.