Amicus Therapeutics Provides Updates on Clinical, Regulatory and Manufacturing Advancements for AT-GAA for Pompe Disease
123 Patients Enrolled in Phase 3 PROPEL Study – Enrollment Completed in 4Q2019
Company Plans to Apply for and Initiate a Rolling Biologics License Application (BLA) for AT-GAA in 2020 with Addition of Full Clinical Results in 1H2021 to Support Full Approval under Fast Track Designation
Promising Innovative Medicine (PIM) Designation Issued by British MHRA with Potential for Early Access for Pompe Patients in
Biologics Manufacturing with
Achieved Full Enrollment of Phase 3 PROPEL Study
During the fourth quarter of 2019, Amicus completed and exceeded patient enrollment in the global Phase 3 PROPEL clinical study for Pompe disease. A total of 59 clinical sites have enrolled 123 patients globally.
PROPEL is a 52-week, double-blind randomized study designed to assess the efficacy, safety and tolerability of AT-GAA compared to the current standard of care, alglucosidase alfa, an enzyme replacement therapy (ERT) in adults with late-onset Pompe disease (LOPD). The PROPEL study is intended to assess superiority of AT-GAA compared to alglucosidase alfa in both ERT switch and ERT naïve patients. The primary efficacy endpoint is change in six-minute walk distance from baseline to Week 52. Secondary endpoints include respiratory measures and additional measures of muscle function and muscle strength. More information, including a list of participating sites, is available at www.clinicaltrials.gov: NCT03729362.
Rolling Biologics License Application (BLA) for AT-GAA
Amicus plans to apply for and initiate a rolling submission of the BLA for AT-GAA in late-onset Pompe disease and to complete final submission in the first half of 2021. A rolling submission allows the Company to submit portions of the regulatory application to the
Promising Innovative Medicine (PIM) designation for AT-GAA
The British Medicines and Healthcare products Regulatory Agency (MHRA) has issued a Promising Innovative Medicine (PIM) designation for AT-GAA in late onset Pompe disease. A PIM designation signifies that a clinical program may be eligible for the Early Access to Medicines Scheme (EAMS), which works to provide patients with life threatening or seriously debilitating conditions access to medicines with the potential to address an unmet medical need.
PIM designation for AT-GAA is based on clinical efficacy results from the Phase 1/2 clinical study, including improvements in six-minute walk distance, an integrated measure of disease progression that evaluates the cardiopulmonary and musculoskeletal systems, as well as comparison to natural history of treated patients. Amicus is evaluating the EAMS program and will provide a future update.
Biologics Manufacturing with
Process performance qualification (PPQ) runs have been initiated with the Company’s key strategic partner,
AT-GAA is an investigational therapy that consists of cipaglucosidase alfa (ATB200), a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake into cells, co-administered with miglustat (AT2221), a pharmacological chaperone. In preclinical studies, AT-GAA was associated with increased tissue enzyme levels, reduced glycogen levels in muscle, and improvements in muscle strength. A global Phase 1/2 study (ATB200-02) is ongoing to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of AT-GAA.
Amicus is also conducting an ongoing global Phase 3 clinical study (ATB200-03, or PROPEL) of AT-GAA in adult patients with late onset Pompe disease. PROPEL is a 52-week, double-blind randomized study designed to assess the efficacy, safety and tolerability of AT-GAA compared to the current standard of care, alglucosidase alfa, an enzyme replacement therapy (ERT). The primary endpoint is six-minute walk distance, an integrated measure of disease progression that evaluates the cardiopulmonary and musculoskeletal systems essential to performing the activities of daily living for patients with Pompe disease. More information, including a list of participating sites, is available at www.clinicaltrials.gov: NCT03729362. In addition, Amicus is enrolling an open-label, uncontrolled, multicenter study to evaluate the PK, safety, efficacy, and PD of AT-GAA in pediatric patients aged 12 to <18 years with LOPD More information, including a list of participating sites, is available at www.clinicaltrials.gov: NCT03911505
About Pompe Disease
Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA levels lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. The disease can be debilitating and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function to a more slowly progressive, late onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.
About Amicus Therapeutics
Director, Investor Relations
Executive Director, Corporate Communications
Source: Amicus Therapeutics, Inc.