Amicus Therapeutics Presents Additional Positive Preliminary Phase 1/2 Data at WORLDSymposium™ 2017
February 15 2017 - 7:30PM
Biomarkers of Muscle Damage (AST, ALT and
CK) Demonstrate Improving Trends in Majority of
Patients
Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company
at the forefront of rare and orphan diseases, today presented
additional positive preliminary data from a global Phase 1/2 study
(ATB200-02) to investigate ATB200/AT2221 in a poster1 at the 13th
Annual WORLDSymposium™ in San Diego, CA. ATB200/AT2221 is a novel
treatment paradigm 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.
“We are very pleased to share these additional
positive preliminary clinical results here at the WORLDSymposium as
we continue with the cascade of data from this important study in
Pompe Disease patients,” said John F. Crowley, Chairman and Chief
Executive Officer of Amicus Therapeutics, Inc. “In addition to
excellent continued safety, tolerability and PK profile, we now see
evidence that this novel Amicus treatment paradigm is further
reducing important biomarkers of disease in both ERT-switch
patients as well as in the first ERT-treatment naïve
patients. In particular, demonstrating with these data that
we can further reduce these biomarkers in a switch population is
very encouraging. To our knowledge, there are no published data for
other Pompe ERTs where these types of reductions in biomarkers have
been shown. Moreover, the fact that these biomarkers have
demonstrated a sustained reduction in a majority of patients over
the first 18 weeks suggests that the effects of our approach may
indeed be persistent and durable. We look forward to data in more
patients and over longer periods of time in the months ahead,
including important measures of muscle function in these patients.
We could not be more pleased with the preliminary data to
date.”
UPDATED CLINICAL DATA
HIGHLIGHTS:
ATB200-02
Clinical Study -- Design and Objectives |
• Primary
objectives: to evaluate safety, tolerability, pharmacokinetics
(PK), and pharmacodynamics (PD) of ATB200/AT2221 |
• Study
duration: 18-week primary treatment period followed by a long-term
extension |
• Patient
cohorts: ambulatory ERT-switch patients (Cohort 1), non-ambulatory
ERT-switch patients (Cohort 2) and ERT-naïve patients (Cohort 3).
Enrolling up to ~20 total patients across all cohorts. |
•
Preliminary data now available: |
• Safety data for 13 patients through interim data analysis
(maximum 36 weeks) |
• PK
and PD (muscle biomarker and disease substrate biomarker) data for
10 patients (eight ERT-switch patients and two naïve patients) |
|
ATB200-02 Study
-- Preliminary Data Highlights in Initial ERT-Switch and
Naive Patients |
|
•
ATB200/AT2221 safety measures (n=13) showed: |
• No
serious adverse events (SAEs) |
• TEAEs were generally mild and transient |
|
•
To date, ATB200/AT2221 has shown no infusion-associated reactions
following 150+ infusions |
|
•
Clinical PK profile was consistent with previously reported
preclinical data (n=10). |
• ATB200
plasma clearance suggests optimized carbohydrate structure provides
efficient uptake into tissues |
• ATB200 alone showed greater than dose-proportional increases
in exposure |
• ATB200 exposure was further enhanced with the addition of
the chaperone AT2221, consistent with stabilization of ATB200 by
AT2221. |
|
•
Reductions observed in biomarkers of muscle damage (creatine kinase
(CK) enzyme, alanine aminotransferase (ALT), and aspartate
aminotransferase (AST)) in ambulatory switch patients (N=8, week
18) and naïve patients (N=2, week 4) |
• In
the eight ERT-switch patients: |
• ALT
decreased in 5 of 8 patients; 4/4 patients with elevated baseline
levels normalized |
• AST
decreased in 6 of 8 patients; 3/4 patients with elevated baseline
levels normalized |
• CK
decreased in 6 of 8 patients; 2/6 patients with elevated baseline
levels normalized |
• ALT,
AST, CK generally remained stable in patients not demonstrating a
decrease |
• In
the two ERT-naïve patients, all three biomarkers improved |
|
•
Reduction observed in a biomarker of glycogen substrate -- Urine
Hexose Tetrasaccharide (Hex4) -- in all eight ERT-switch patients
and both naïve patients; overall reduction approximately 30%
|
|
•
Next interim analysis anticipated in the coming
months |
|
About ATB200/AT2221ATB200/AT2221 is a novel
treatment paradigm 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 preclinical studies, ATB200 was associated with
increased tissue enzyme levels and reduced glycogen levels in
muscle, which was further improved when AT2221 was co-administered
with ATB200. Amicus Therapeutics is currently conducting a global
Phase 1/2 study (ATB200-02) to evaluate the safety, tolerability,
pharmacokinetics (PK) and pharmacodynamics of ATB200/AT2221.
About Pompe DiseasePompe
disease is an inherited lysosomal storage disorder caused by
deficiency of an enzyme called acid alpha-glucosidase (GAA).
Reduced or absent levels of GAA lead to the accumulation of the
substrate glycogen in the lysosomes of muscles and other tissues.
Progressive accumulation of glycogen is believed to lead to the
morbidity and mortality associated with Pompe disease, including
muscle weakness and respiratory insufficiency.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a global biotechnology company
at the forefront of therapies for rare and orphan diseases. The
Company has a robust pipeline of advanced therapies for a broad
range of human genetic diseases. Amicus’ lead programs in
development include the small molecule pharmacological chaperone
migalastat as a monotherapy for Fabry disease, SD-101 for
Epidermolysis Bullosa (EB), as well as novel enzyme replacement
therapy (ERT) and biologic products for Fabry disease, Pompe
disease, and other rare and devastating diseases.
1Johnson, et. al, WORLDSymposium
2017, First-in-human preliminary pharmacokinetic and
safety data on a novel recombinant acid-α-glucosidase, ATB200,
co-administered with the pharmacological chaperone, AT2221, in
ERT-experienced Pompe patients
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 encouraging preliminary data from
a global phase 1/2 study to investigate ATB200/AT2221 for the
treatment of Pompe and the potential implications on these
data for the future advancement and development of ATB200/AT2221.
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
the preliminary data 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 ATB200/AT2221, 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 ATB200/AT2221. 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, 2015 and Quarterly Report on 10-Q for the Quarter
ended September 30, 2016. 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.
FOLD–G
CONTACTS:
Investors/Media:
Amicus Therapeutics
Sara Pellegrino
Senior Director, Investor Relations
spellegrino@amicusrx.com
(609) 662-5044
Media:
MWW PR
Sid Dinsay
sdinsay@mww.com
(646) 381-9017
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