Here Are Amicus’s Key Research Programs in 2019


May. 23 2019, Published 12:08 p.m. ET

Vision for fiscal 2023

In its investor presentation, Amicus Therapeutics (FOLD) has guided for revenues of around $200 million and $300 million for its AT-GAA Novel ERT (enzyme replacement therapy) and portfolio of gene therapy and inlicensed products, respectively, for fiscal 2023. The company expects to have successfully completed clinical trials and secured regulatory approvals for these products in fiscal 2023. Additionally, Amicus Therapeutics expects to have more than five research programs in clinic and the ability to file for more than one IND (initial new drug) application every 12 to 18 months by fiscal 2023.

Amicus Therapeutics is targeting a market opportunity estimated at $1.0 billion to $2.0 billion with its investigational AT-GAA ERT in the Pompe disease indication, and a market opportunity estimated at $1.0 billion with its investigational gene therapy products.

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AT-GAA in Pompe disease

On February 5, Amicus Therapeutics issued a press release announcing positive data from the Phase 1/2 trial evaluating investigational AT-GAA therapy administered for up to 24 months, in the late-onset Pompe disease indication. On February 25, the company issued a press release announcing the grant of BTD (breakthrough therapy designation) by FDA to AT-GAA therapy in late-onset Pompe disease indication.

Amicus Therapeutics expects to treat more than 100 Pompe patients with its AT-GAA therapy by end of fiscal 2019. According to the first-quarter earnings conference call, the company expects data readout from the fourth cohort of Phase 1 / 2 study for AT-GAA therapy and natural history data for Pompe disease patients who are on FDA approved ERT (enzyme replacement therapy) in fiscal 2019.

Amicus Therapeutics is also enrolling patients in the pivotal PROPEL trial for AT-GAA therapy in the Pompe disease indication across 24 sites. The company plans to open up 90 enrollment sites around the world, and include both ERT (enzyme replacement therapy) naïve and ERT switch patients in this trial.

Amicus Therapeutics aims to position AT-GAA therapy as a standard of care in the Pompe disease indication.


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