Amicus Therapeut Stock Price, News & Analysis

Amicus Therapeutics (Nasdaq: FOLD) presented new 4-year data from the PROPEL open-label extension study of Pombiliti® + Opfolda® in adults with late-onset Pompe disease (LOPD). The analysis focused on 82 patients, including 62 ERT-experienced and 20 ERT-naïve individuals.

Key findings in ERT-experienced patients after 208 weeks showed improvements across multiple metrics: +2.3% in six-minute walk distance, +1.6 points in lower extremity manual muscle test score, and notable improvements in other functional measures. The treatment demonstrated a favorable safety profile with no new safety signals identified, though 41 patients experienced treatment-related adverse events.

The data supports the durability and efficacy of Pombiliti + Opfolda in treating LOPD patients who are not improving on current enzyme replacement therapy (ERT).

Amicus Therapeutics (Nasdaq: FOLD), a global biotechnology company focused on rare diseases, has announced its participation in two major healthcare investor conferences in September 2025. The company will present at the Cantor Global Healthcare Conference on September 3 at 10:20 a.m. ET and the Morgan Stanley 23rd Annual Global Healthcare Conference on September 9 at 3:20 p.m. ET.

Both presentations will be accessible via live audio webcast through the investor relations section of Amicus Therapeutics' corporate website. The company continues its commitment to developing first- and best-in-class medicines for rare diseases.

Amicus Therapeutics (Nasdaq: FOLD) reported strong Q2 2025 financial results with total revenue of $154.7M, up 18% at constant exchange rates. The company's performance was driven by two commercial therapies: Galafold revenue reached $128.9M (up 12%) and Pombiliti + Opfolda achieved $25.8M (up 58%).

Key highlights include Japanese regulatory approval for Pombiliti + Opfolda in adult LOPD patients, progress in the ACTION3 Study of DMX-200 for FSGS, and anticipated GAAP profitability in H2 2025. The company maintains its 2025 guidance with projected revenue growth of 15-22% and expects to exceed $1 billion in total revenue by 2028.

Q2 GAAP net loss was $24.4M ($0.08 per share), while non-GAAP net income was $1.9M ($0.01 per share). Cash position stood at $231.0M as of June 30, 2025.

Amicus Therapeutics (Nasdaq: FOLD), a global biotechnology company focused on rare diseases, will host a conference call and audio webcast on Thursday, July 31, 2025, at 8:30 a.m. ET to discuss its second quarter 2025 financial results.

Participants must pre-register online to receive dial-in details and a personal PIN for the call. A live webcast and presentation materials will be available through the Investors section at ir.amicusrx.com, with web participants encouraged to register 15 minutes before the call. An archived version of the webcast and slides will be available after the event.

Amicus Therapeutics (Nasdaq: FOLD) has received approval from Japan's Ministry of Health, Labour and Welfare (MHLW) for Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) to treat adult patients with late-onset Pompe disease (LOPD). The approval was based on the Phase 3 PROPEL study, which uniquely evaluated both ERT-naïve and ERT-experienced participants.

This two-component therapy consists of Pombiliti, a recombinant human GAA enzyme designed for increased muscle cell uptake, and Opfolda, an enzyme stabilizer. The treatment is now approved across major markets including the U.S., E.U., U.K., Canada, Australia, Switzerland, and Japan.

Amicus Therapeutics announced new post-hoc analysis results from the PROPEL study of Pombiliti + Opfolda in late-onset Pompe disease (LOPD), published in Muscle and Nerve. The analysis focused on ERT-experienced patients who switched from alglucosidase alfa to cipaglucosidase alfa-atga + miglustat (cipa+mig). Results showed that patients who switched to cipa+mig (n=65) demonstrated improvements or stability across multiple outcomes, including 6-minute walk distance, muscle testing, and PROMIS-Fatigue scores. In contrast, patients who remained on alglucosidase alfa + placebo (n=30) generally showed worsening or stability. The PROPEL study involved 123 adult LOPD patients, with 77% previously treated with alglucosidase alfa. The findings suggest potential benefits for LOPD patients switching to Pombiliti + Opfolda, particularly in maintaining or improving various functional measures in this progressive condition.

Amicus Therapeutics (FOLD) has announced its participation in two major investor conferences in June 2025. The company will present at the Jefferies Global Healthcare Conference in New York on June 4 at 11:40 a.m. ET and at the Goldman Sachs 46th Annual Global Healthcare Conference in Miami on June 10 at 2:00 p.m. ET. Both presentations will be accessible via live audio webcast through the investor relations section of Amicus' corporate website. Amicus Therapeutics is a global biotechnology company dedicated to developing and delivering innovative medicines for rare diseases, with a strong focus on patient care and advancing their pipeline of first- or best-in-class treatments.

Amicus Therapeutics (FOLD) has announced its participation in the Bank of America 2025 Health Care Conference in Las Vegas. The company's management will engage in a fireside chat on Wednesday, May 14, 2025, at 8:00 a.m. PT. Investors and interested parties can access a live audio webcast of the presentation through the investors section of Amicus Therapeutics' corporate website.

Amicus Therapeutics is a global biotechnology company dedicated to discovering, developing, and delivering innovative medicines for rare diseases, with a strong focus on patient care and advancing their pipeline of first- or best-in-class treatments.

Amicus Therapeutics (FOLD) reported Q1 2025 total revenue of $125.2M, marking a 15% year-over-year increase at constant exchange rates. The company's portfolio includes Galafold with sales of $104.2M (+6% YoY) and Pombiliti + Opfolda with $21.0M (+92% YoY). Amicus updated its 2025 guidance, adjusting total revenue growth to 15-22% and maintaining its path to GAAP profitability in H2 2025. The company expanded its portfolio by in-licensing DMX-200, a Phase 3 program for rare kidney disease FSGS. The company reported a GAAP net loss of $21.7M ($0.07 per share) and non-GAAP net income of $9.0M ($0.03 per share). Cash position remained stable at $250.6M as of March 31, 2025.