Amicus Therapeutics (Nasdaq: FOLD), a global, patient-dedicated
biotechnology company focused on discovering, developing and
delivering novel medicines for rare metabolic diseases, today
announced clinical, regulatory and manufacturing advancements for
AT-GAA, its investigational therapy for Pompe disease. Pompe
disease is an inherited lysosomal disorder caused by an enzyme
deficiency that leads to accumulation of glycogen (disease
substrate) in cells and is characterized by severe muscle weakness
that worsens over time.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics stated, “Our team’s efforts to bring
AT-GAA to all patients living with Pompe around the world continues
at a momentous pace and with great passion. We know that there is
an urgent need for new, second-generation therapies for people
living with lysosomal disorders, especially in Pompe disease.
Regulatory agencies in the U.S. and now the U.K., have recognized
the highly differentiated clinical data set and the great potential
for AT-GAA to meet the currently unmet medical needs in Pompe
disease through the granting of Breakthrough Therapy Designation
and the Promising Innovative Medicine Designation, respectively.
Amicus now plans to apply for and initiate a rolling submission of
the AT-GAA BLA in the U.S. later this year. These designations,
together with the results from the Phase 2 study and the now fully
enrolled PROPEL pivotal study, support our strategy to advance
AT-GAA as quickly as possible with the potential to become the new
standard of care for all persons living with Pompe disease.”
Achieved Full Enrollment of Phase 3
PROPEL StudyDuring 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-GAAAmicus 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 U.S. Food and Drug Administration
(FDA) as they are completed, rather than waiting until every
section of the BLA is complete to submit the entire application for
review.
The U.S. FDA previously granted Breakthrough
Therapy Designation to AT-GAA for the treatment of late onset Pompe
disease 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.
Promising Innovative Medicine (PIM)
designation for AT-GAAThe 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 WuXi
Biologics on TrackProcess performance qualification (PPQ)
runs have been initiated with the Company’s key strategic partner,
WuXi Biologics. The PPQ runs will serve as the foundation for the
Chemistry, Manufacturing, and Control (CMC) module for a BLA
submission. The manufacturing focus remains on AT-GAA inventory
build, which will support: the clinical studies underway, further
studies including in pediatrics, as well as to supply the early
commercial inventory. WuXi Biologics will enable and support Amicus
with its dual sourcing strategy and manufacture at two sites across
their network, including its new facility in Ireland, which remains
on-track.
About AT-GAAAT-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 DiseasePompe 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 TherapeuticsAmicus
Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated
biotechnology company focused on discovering, developing and
delivering novel high-quality medicines for people living with rare
metabolic diseases. With extraordinary patient focus, Amicus
Therapeutics is committed to advancing and expanding a robust
pipeline of cutting-edge, first- or best-in-class medicines for
rare metabolic diseases. For more information please visit the
company’s website at www.amicusrx.com, and follow us
on Twitter and LinkedIn.
CONTACTS:
Investors:
Andrew FaughnanDirector, Investor
Relationsafaughnan@amicusrx.com (609) 662-3809
Media:Christopher Byrne Executive Director,
Corporate Communications cbyrne@amicusrx.com (609) 662-2798
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