Amicus Therapeutics Issued Two U.S. Patents for Pompe Enzyme ATB200
March 20 2019 - 7:00AM
Amicus Therapeutics (Nasdaq: FOLD) today announced that the United
States Patent and Trademark Office has issued two new patents
directed to the composition of matter and methods of making ATB200,
a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme
with optimized carbohydrate structures, particularly mannose-6
phosphate (M6P), to enhance uptake. ATB200 is the biologic
component of the Company’s investigational Pompe treatment paradigm
AT-GAA, which consists of ATB200 co-administered with AT2221, a
pharmacological chaperone.
The first U.S. patent No. 10,208,299 reflects
composition of matter for highly potent rhGAA with enhanced
carbohydrates. The second U.S. patent No. 10,227,577 covers the
methods for making ATB200. The patents are set to expire in 2035
and 2036, respectively. Amicus is pursuing corresponding patent
applications in other regions and countries, including Europe and
Japan.
“These newly issued U.S. patents reflect the
novelty and uniqueness of our protein engineering and biologics
expertise to develop a differentiated, highly potent recombinant
GAA enzyme as the key component of our novel Pompe treatment
paradigm,” said John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics. “We believe these attributes are
necessary to optimally target this enzyme to muscles in patients.
The issuance of these key patents also embodies the many years of
tremendous hard work, perseverance and ingenuity of the Amicus
science and technical operations teams as well as the enormous
investment of capital required to create and make this new
medicine. These patents provide Amicus with broad and long-term
intellectual property rights into the mid-2030s and significantly
strengthen our position as we advance AT-GAA to become the next
potential standard of care for Pompe disease. As we continue to
serve patients globally and invest in additional research and
development for AT-GAA, we also look forward to further expanding
our intellectual property portfolio around the world.”
About AT-GAA
AT-GAA is an investigational therapy that
consists of ATB200, a unique recombinant human acid
alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate
structures, particularly mannose-6 phosphate (M6P), to enhance
uptake, co-administered with AT2221, a pharmacological chaperone.
In 2019, the U.S. Food and Drug Administration (FDA) granted
Breakthrough Therapy Designation to AT-GAA for the treatment of
late onset Pompe disease. 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 currently sponsoring PROPEL, a global
Phase 3 clinical study (also known as ATB200-03) 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).
More information, including a list of participating sites, is
available at www.clinicaltrials.gov: NCT03729362.
About Pompe DiseasePompe
disease is an inherited lysosomal storage disorder caused by
deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or
absent levels of GAA leads 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
Amicus 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.
Forward-Looking StatementsThis
press release contains "forward- looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995,
including statements relating to patent scope and length of
protection, encouraging preliminary data from a global Phase 1/2
study to investigate AT-GAA for the treatment of Pompe and the
potential implications on these data for the future advancement and
development of AT-GAA. Words such as, but not limited to, “look
forward to,” “believe,” “expect,” “anticipate,” “estimate,”
“intend,” "confidence," "encouraged," “potential,” “plan,”
“targets,” “likely,” “may,” “will,” “would,” “should” and “could,”
and similar expressions or words identify forward-looking
statements. The forward looking statements included in this press
release are based on management's current expectations and belief's
which are subject to a number of risks, uncertainties and
factors, including that biosimilars or others could challenge
the validity of the AT-GAA patents, Amicus may not be able to
enforce the patents, there could be patent-related litigation, the
preliminary data from the Ph 1/2 study is based on a small patient
sample and reported before completion of the study will
not be predictive of future results, that results of
additional preliminary data or data from the completed study
or any future study will not yield results that are consistent with
the preliminary data presented, that the Company will be not
able to demonstrate the safety and efficacy of AT-GAA, that
later study results will not support further
development, or even if such later results are favorable,
that the Company will not be able to successfully complete
the development of, obtain regulatory approval for, or successfully
commercialize AT-GAA. In addition, all forward looking
statements are subject to the other risks and uncertainties
detailed in our Annual Report on Form 10-K for the year ended
December 31, 2018. As a consequence, actual results may differ
materially from those set forth in this press release. You
are cautioned not to place undue reliance on these forward looking
statements, which speak only of the date hereof. All forward
looking statements are qualified in their entirety by this
cautionary statement and we undertake no obligation to revise this
press release to reflect events or circumstances after the date
hereof.
CONTACTS:
Investors/Media:Amicus TherapeuticsSara
Pellegrino, IRCVice President, Investor Relations & Corporate
Communicationsspellegrino@amicusrx.com(609) 662-5044
Media:Amicus TherapeuticsMarco
Winkler Director, Corporate Communications
mwinkler@amicusrx.com(609) 662-2798
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