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Amicus Therapeutics, Inc. (symbol: FOLD) is a global biotechnology company that specializes in discovering, developing, and delivering high-quality medicines for individuals suffering from rare metabolic diseases. The company's central focus revolves around addressing lysosomal storage disorders, such as Fabry disease and Pompe disease, through its innovative platform technologies and medicines.
Amicus Therapeutics operates with a patient-centric approach, ensuring that the needs of those in the rare disease community are at the forefront of their scientific research, commercial activities, and clinical programs. The company is dedicated to creating significant impacts on the lives of patients and their caregivers, striving to provide advanced therapeutic solutions where none previously existed.
The company's product pipeline includes:
- Galafold® (migalastat): An oral chaperone therapy approved for the treatment of adults with Fabry disease who have amenable genetic variants. Galafold works by stabilizing the body’s own dysfunctional enzyme to clear the accumulated substrate.
- Pombiliti™ and Opfolda™: A novel, two-component therapy designed for the treatment of late-onset Pompe disease, showcasing Amicus's commitment to addressing the complexities of metabolic disorders.
- Rare Disease Gene Therapy Portfolio: A set of advanced gene therapies aimed at providing long-lasting solutions for various rare metabolic diseases.
Amicus Therapeutics is focused on expanding its pipeline with first-in-class or best-in-class treatments, underscoring its commitment to innovative medical solutions. Financially, the company maintains a strong position, actively pursuing strategic priorities to sustain growth and enhance shareholder value.
With a presence in 27 countries, Amicus Therapeutics leverages a global footprint to address unmet medical needs across diverse patient populations. The company continuously seeks opportunities to broaden its impact and foster collaborations that advance its mission.
Recent developments include regulatory approvals and strategic partnerships aiming to enhance the reach and effectiveness of their therapies. By building strong relationships with stakeholders, Amicus Therapeutics ensures a collaborative effort in combating rare diseases.
Overall, Amicus Therapeutics Inc. embodies a profound dedication to transforming the lives of those affected by rare diseases through innovative science, patient-focused care, and robust therapeutic development.
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.
Amicus Therapeutics (FOLD) reported Galafold revenue of approximately $261 million for 2020, exceeding guidance of $250-$260 million and reflecting a 43% year-over-year growth. The company completed the last patient visit in the AT-GAA Phase 3 PROPEL study, with data expected in 1Q2021. Amicus anticipates Galafold revenue of at least $300 million for 2021 and plans to submit the BLA for AT-GAA in the first half of the year. With a strong cash position, the company aims for self-sustainability without further dilutive financing.
Amicus Therapeutics (Nasdaq: FOLD) announced that its CEO, John F. Crowley, will present at the 39th Annual J.P. Morgan Healthcare Conference on January 12, 2021, at 8:20 a.m. ET. A live audio webcast of the presentation will be available on the company’s investor website.
Amicus Therapeutics focuses on developing innovative therapies for rare metabolic diseases, driven by a commitment to patient care. For insights, visit the company's website.
Amicus Therapeutics (Nasdaq: FOLD) has been awarded the Great Place to Work-Certified™ status for 2020, recognizing its positive workplace culture as rated by employees. The certification is based on validated feedback and highlights Amicus's commitment to employee experience and corporate culture. Chief People Officer David Clark emphasized the company's focus on diverse backgrounds and employee engagement to foster talent and innovation. This accolade indicates a strong alignment between workplace culture and business performance, which may positively influence investor confidence.
Amicus Therapeutics (Nasdaq: FOLD) has appointed Michael A. Kelly to its Board of Directors, bringing over 20 years of experience in the life sciences sector. Kelly, who founded Sentry Hill Partners, previously held senior positions at Amgen, including CFO. CEO John Crowley highlighted Kelly's expertise as vital for the company's growth, particularly in navigating the biotechnology landscape focused on rare diseases. Kelly expressed enthusiasm to contribute to Amicus's mission during a pivotal growth phase.
Amicus Therapeutics (Nasdaq: FOLD) announced the initiation of its rolling Biologics License Application (BLA) submission to the FDA for AT-GAA, a therapy for late onset Pompe disease (LOPD). This rolling submission allows the company to submit application sections as they are completed. The FDA granted Breakthrough Therapy Designation for AT-GAA based on positive Phase 1/2 trial results. Top-line data from the global Phase 3 PROPEL study is expected in 1Q2021, with all manufacturing activities completed successfully.
Amicus Therapeutics (Nasdaq: FOLD) will participate in a fireside chat at the Evercore ISI 3rd Annual HealthCONx Conference on December 1, 2020, at 2:15 p.m. E.T. Interested parties can listen to the event through a live audio webcast available on the company’s corporate website. Amicus is dedicated to developing innovative treatments for rare metabolic diseases, reflecting its commitment to patient care and a robust pipeline of novel therapies. For more details, visit their website.
Amicus Therapeutics (Nasdaq: FOLD) reported 3Q20 revenue of $67.4 million, a 38% increase from $48.8 million in 3Q19, affirming its full-year revenue guidance of $250 million to $260 million. The revenue growth is mainly driven by strong demand for Galafold, with compliance rates exceeding 90%. The PROPEL study for AT-GAA is on track for data release in 1Q21, while positive CLN6 gene therapy data has been presented. The company maintains a strong cash position of $509.1 million, supporting ongoing operations through to self-sustainability.
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