Amicus Therapeut Stock Price, News & Analysis

Amicus Therapeutics (Nasdaq: FOLD) has appointed Dr. Eiry W. Roberts to its Board of Directors. Dr. Roberts brings nearly 30 years of drug development experience, having previously served as Chief Medical Officer at Neurocrine Biosciences and held senior roles at Eli Lilly. She has led numerous late-stage development programs to global regulatory approvals, contributing significantly to her former company’s portfolio. Her expertise in healthcare strategy is expected to enhance Amicus’s mission of developing therapies for rare diseases.

Amicus Therapeutics (FOLD) announced that the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) granted a positive scientific opinion for AT-GAA under the Early Access to Medicines Scheme. This allows eligible adults with late-onset Pompe disease to access AT-GAA prior to marketing authorization. The decision underscores the high unmet medical need in this patient group. AT-GAA, a two-component therapy, has shown promising results in clinical studies. Marketing Authorization Applications for AT-GAA are expected in the UK and EU in late 2021.

Amicus Therapeutics (Nasdaq: FOLD) will participate in the Goldman Sachs 42nd Annual Global Healthcare Conference on June 9, 2021, at 5:30 p.m. E.T. The event will include a fireside chat featuring members of Amicus' senior leadership team. Investors can listen to the live audio webcast via the company’s investor relations page. Amicus focuses on developing innovative medicines for rare metabolic diseases, underscoring its commitment to patient care and advancing a strong pipeline of therapies.

Amicus Therapeutics (Nasdaq: FOLD) announced the presentation of two posters at the virtual 16th International Congress on Neuromuscular Diseases on May 21-22 and 28-29, 2021. The posters focus on its development program for Pompe disease, including:

• Enhancing Delivery of Acid Alpha-Glucosidase to Skeletal Muscle by Nithya Selvan, Ph.D.
• Efficacy and Safety of Cipaglucosidase Alfa/Miglustat versus Alglucosidase Alfa/Placebo in Late-Onset Pompe Disease presented by Benedikt Schoser, MD.

The posters will be available on the Amicus website after the presentations.

Amicus Therapeutics reported Q1 2021 revenue of $66.4 million for Galafold, a 9.8% increase year-over-year, and remains on track to meet its full-year guidance of $300M-$315M. Patient demand for Galafold has risen, and compliance exceeds 90%. The company is progressing with the rolling BLA submission for AT-GAA in Pompe disease, anticipating completion in Q2 2021. Amicus maintains a cash position of $417.4 million, sufficient for operations through to self-sustainability.

Amicus Therapeutics (Nasdaq: FOLD) announced participation in two upcoming virtual investor conferences in May 2021. The Bank of America 2021 Health Care Conference is scheduled for May 12 at 1:15 p.m. E.T., followed by the UBS Global Healthcare Virtual Conference on May 24 at 10:00 a.m. E.T.. Investors can access live webcasts of these presentations on the company’s corporate website. Amicus Therapeutics is dedicated to developing innovative therapies for rare metabolic diseases.

Amicus Therapeutics (Nasdaq: FOLD) has appointed Sébastien Martel as Senior Vice President of Strategy and Business Development. With 25 years in the pharmaceutical and biotechnology sectors, Martel previously led Global Rare Diseases at Sanofi. His expertise encompasses corporate strategy and commercialization in rare diseases. Amicus CEO John F. Crowley highlighted Martel's experience as crucial for advancing the company's growth strategies and enhancing its portfolio of rare disease therapeutics.

Amicus Therapeutics (Nasdaq: FOLD) has announced the acceptance of two abstracts for the American Society of Gene & Cell Therapy 24th Annual Meeting, occurring virtually from May 11-14, 2021. The presentations will feature preclinical data from the company's gene therapy programs targeting Fabry and Pompe diseases. The Pompe disease abstract, presented by Ali Ramezani, focuses on muscle pathology reversal, while the Fabry disease abstract, by CJ Song, discusses the efficacy of a new transgene. These insights underscore Amicus's commitment to rare metabolic diseases.

Amicus Therapeutics (NASDAQ:FOLD) announced the successful completion of a Type B Pre-Biologics License Application (BLA) meeting with the FDA for its investigational therapy AT-GAA, aimed at treating Pompe disease. They are on track to complete the rolling BLA submission by the end of Q2 2021, including data from Phase 1/2 and Phase 3 studies. AT-GAA has previously received Breakthrough Therapy Designation from the FDA. The company plans to submit marketing applications to the EMA in the latter half of 2021, focusing on improving the lives of Pompe disease patients.