Strong Momentum for Galafold Launch in
Europe and Multiple Global Regulatory Submissions
Ahead
Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company
at the forefront of therapies for rare and orphan diseases, today
provided its full-year 2017 strategic outlook and financial
guidance.
“In 2016 we made significant progress in our
transformation to a global commercial-stage biotech company while
we continued to advance and expand our tremendous pipeline of
first- and/or best-in-class medicines for people living with
devastating rare diseases,” stated John F. Crowley, Chairman and
Chief Executive Officer of Amicus Therapeutics, Inc. “We begin 2017
in an excellent position to develop and deliver great medicines for
patients and to create significant shareholder value. For this
year, we are laser focused on five key strategic priorities: 1)
advancing the international Galafold launch, 2) completing our
regulatory submission for migalastat in Japan, 3) establishing our
novel Pompe treatment paradigm ATB200/AT2221 as a highly
differentiated therapy, 4) successfully completing our Phase 3
clinical study in patients with epidermolysis bullosa, and 5)
maintaining our financing strength. With one commercial-stage
medicine and two medicines in mid- and late-stage clinical
development, as well as a biologics platform for future growth, our
vision is to become a leading global biotechnology company focused
on delivering meaningful benefits for patients with devastating
rare diseases.”
Key 2016 Accomplishments
- Galafold™ (migalastat) full approval in European Union
(EU) – first precision medicine and first oral treatment
for Fabry disease received a broad label for patients 16 years and
older with a confirmed diagnosis of Fabry disease and who have an
amenable genetic mutation (estimated 35%-50% of the global Fabry
population)
- Galafold international launch success –
initial launch exceeding expectations with 61 patients on
reimbursed Galafold (commercial or expanded access programs, or
EAPs) as of December 31, 2016
- ATB200/AT2221 positive preliminary data from Pompe
clinical study – compelling data on safety and
pharmacokinetic (PK) profile in addition to biomarkers of muscle
damage set important foundation for differentiating ATB200/AT2221
from any other approach
- Enrollment near complete in Phase 3 epidermolysis
bullosa (EB) Study – significant momentum with sites now
active in the U.S., Europe, and Australia
- Balance sheet strengthened - cash, cash
equivalents, and marketable securities totaled $331 million at
December 31, 2016
Mr. Crowley will discuss Amicus' corporate
objectives and key milestones in a presentation at the 35th Annual
J.P. Morgan Healthcare Conference on Tuesday, January 10, 2017 at
8:30 a.m. PT (11:30 a.m. ET). A live webcast of the presentation
can be accessed through the Investors section of the Amicus
Therapeutics corporate web site at
http://ir.amicusrx.com/events.cfm, and will be archived for 90
days.
2017 Financial Guidance
Cash, cash equivalents, and marketable
securities totaled $331 million at December 31, 2016. As previously
announced, the Company strengthened the balance sheet during 2016
with a $100 million at-the-market (ATM) equity financing as well as
a $250 million convertible debt offering. The Company expects
full-year 2017 net operating cash flow of between $175 million to
$200 million and expects full-year 2017 total net cash spend
(including third-party milestone payments and capital expenditures)
of between $200 million and $225 million.
Program Highlights
Migalastat for Fabry
Disease
Migalastat is an oral precision medicine
intended to treat Fabry disease in patients who have amenable
genetic mutations. As previously announced, the European Commission
(EC) has granted full approval for migalastat under the trade name
Galafold. The EC approval may serve as the basis for regulatory
approvals in more than two-thirds of the global Fabry market that
is outside the U.S. The Company has also defined a U.S. pathway to
support full approval.
International Launch and Expanded Access
Programs (EAP) Updates:
- 61 patients (naïve and ERT-switch) on reimbursed Galafold as of
December 31, 2016
- Six countries with reimbursement (commercial or EAP)
- Reimbursement dossiers submitted and pricing discussions are
now underway in 18 countries
- National Institute for Health and Care Excellence (NICE) issued
a final positive recommendation for reimbursement of Galafold in
England
- Target of 300 patients treated with reimbursed Galafold by
year-end 2017
Regulatory Updates:
- One additional approval secured outside EU (Switzerland)
- Regulatory submissions completed in six additional territories
outside EU
- U.S. regulatory pathway defined by Amicus to support full
approval
Anticipated Upcoming Fabry Disease Program Milestones:
- EU commercial launch in additional countries and EAP in
additional territories
- Additional regulatory submissions including a Japanese
regulatory submission (J-NDA) targeted for 1H17
- U.S. intermediate EAP
- Fabry ERT cell line development and program update
- Pivotal gastrointestinal (GI) symptoms study initiation
ATB200/AT2221 for Pompe
Disease
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.
Positive preliminary data were reported in the fourth quarter of
2016 from a global clinical study (ATB200-02) to evaluate safety,
tolerability, PK, and pharmacodynamics (PD) of ATB200/AT2221. The
study is enrolling 3 cohorts of patients, including ambulatory
ERT-switch patients (Cohort 1), non-ambulatory ERT-switch patients
(Cohort 2), and ERT-naïve patients (Cohort 3).
Key Preliminary Data Highlights from ATB200-02
Study in Initial ERT-Switch Patients:
- No infusion-associated reactions following 100+ infusions in
nine initial ERT-switch patients treated for a maximum of 24
weeks
- Available PK and PD (muscle biomarker) data through week 14 in
four initial ERT-switch patients showed the desired PK profile and
improvements in key muscle damage biomarkers in two of four
patients
Anticipated Upcoming Pompe Disease Program
Milestones:
- Additional ATB200-02 study clinical data as well as new
preclinical findings to be presented at the 13th Annual
WORLDSymposium in San Diego, CA from February 13-17, 2017
- ATB200-02 study data in naïve and non-ambulatory patients, as
well as extension-phase data on all patient cohorts
- Meetings with US and EU regulators
SD-101 for Epidermolysis Bullosa
(EB)
SD-101 is a novel, late-stage, proprietary
topical treatment and potential first-to-market therapy for EB.
SD-101 is currently being investigated in a registration-directed
Phase 3 study (ESSENCE, also known as SD-005) to support global
regulatory submissions.
SD-101 was granted FDA Breakthrough Therapy
designation in 2013 based on results from a Phase 2a study for the
treatment of lesions in patients suffering with EB. SD-101 is the
first-ever treatment in clinical studies to show improvements in
wound closure across all major EB types.
EB Phase 3 ESSENCE Study Highlights:
- Significant momentum enrolling patients diagnosed with Simplex,
Recessive Dystrophic, or Junctional non-Herlitz EB
- More than 95% of patients completing the primary treatment
period have elected to continue in the open-label extension
study
Anticipated EB Program Milestones:
- Top-line Phase 3 data anticipated mid-2017
Important Safety
InformationTreatment with GALAFOLD should be initiated and
supervised by specialists experienced in the diagnosis and
treatment of Fabry disease. GALAFOLD is not recommended for use in
patients with a nonamenable mutation.
- GALAFOLD is not intended for concomitant use with enzyme
replacement therapy.
- GALAFOLD is not recommended for use in patients with Fabry
disease who have severe renal impairment (<30 mL/min/1.73 m2).
The safety and efficacy of GALAFOLD in children 0–15 years of age
have not yet been established.
- No dosage adjustments are required in patients with hepatic
impairment or in the elderly population.
- There is very limited experience with the use of this medicine
in pregnant women. If you are pregnant, think you may be pregnant,
or are planning to have a baby, do not take this medicine until you
have checked with your doctor, pharmacist, or nurse.
- While taking GALAFOLD, effective birth control should be used.
It is not known whether GALAFOLD is excreted in human milk.
- Contraindications to GALAFOLD include hypersensitivity to the
active substance or to any of the excipients listed in the
PRESCRIBING INFORMATION.
- It is advised to periodically monitor renal function,
echocardiographic parameters and biochemical markers (every 6
months) in patients initiated on GALAFOLD or switched to
GALAFOLD.
- OVERDOSE: General medical care is recommended in the case of
GALAFOLD overdose.
- The most common adverse reaction reported was headache, which
was experienced by approximately 10% of patients who received
GALAFOLD. For a complete list of adverse reactions, please review
the SUMMARY OF PRODUCT CHARACTERISTICS.
- Call your doctor for medical advice about side effects.
For further important safety information for Galafold, including
posology and method of administration, special warnings, drug
interactions and adverse drug reactions, please see the European
SmPC for Galafold available from the EMA website at
www.ema.europa.eu.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a 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.
Forward-Looking StatementsThis
press release contains "forward-looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995
relating to preclinical and clinical development of our product
candidates, the timing and reporting of results from preclinical
studies and clinical trials, the prospects and timing of the
potential regulatory approval of our product candidates,
commercialization plans, financing plans, and the projected cash
position for the Company. In particular, this press release relates
to the preliminary data from a global Phase 1/2 study (ATB200-02)
to investigate ATB200/AT2221. The inclusion of forward-looking
statements arising from this preliminary data and study should not
be regarded as a representation by us that any of our plans will be
achieved. Any or all of the forward-looking statements in this
press release may turn out to be wrong and can be affected by
inaccurate assumptions we might make or by known or unknown risks
and uncertainties. For example, with respect to statements
regarding the goals, progress, timing, and outcomes of discussions
with regulatory authorities, and in particular the potential goals,
progress, timing, and results of preclinical studies and clinical
trials, actual results may differ materially from those set forth
in this release due to the risks and uncertainties inherent in our
business, including, without limitation: the potential that results
of clinical or preclinical studies indicate that the product
candidates are unsafe or ineffective; the potential that it may be
difficult to enroll patients in our clinical trials; the potential
that regulatory authorities, including the FDA, EMA, and PMDA, may
not grant or may delay approval for our product candidates; the
potential that we may not be successful in commercializing Galafold
in Europe or our other product candidates if and when approved; the
potential that preclinical and clinical studies could be delayed
because we identify serious side effects or other safety issues;
and the potential that we will need additional funding to complete
all of our studies. Further, the results of earlier preclinical
studies and/or clinical trials may not be predictive of future
results. The preliminary data and Phase 1/2 study discussed herein
is inherently preliminary and early in the study, derived from a
limited patient set, and later trial results with this patient set
or others may not be consistent with these preliminary results.
With respect to statements regarding projections of the Company's
cash position, actual results may differ based on market factors
and the Company's ability to execute its operational and budget
plans. In addition, all forward-looking statements are subject to
other risks detailed in our Annual Report on Form 10-K for the year
ended December 31, 2015 and Quarterly Report on Form 10-Q for the
quarter ended September 30, 2016. You are cautioned not to place
undue reliance on these forward-looking statements, which speak
only as of the date hereof. All forward-looking statements are
qualified in their entirety by this cautionary statement, and we
undertake no obligation to revise or update this news 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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