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8-K - FORM 8-K - AMICUS THERAPEUTICS, INC. | c26618e8vk.htm |
EX-99.1 - EXHIBIT 99.1 - AMICUS THERAPEUTICS, INC. | c26618exv99w1.htm |
Exhibit 99.2
Amicus Therapeutics Provides Full-Year 2012
Strategic Outlook and Financial Guidance
Strategic Outlook and Financial Guidance
Phase 3 Results of Migalastat HCl Monotherapy for Fabry Disease on Track for 3Q12
Positive Preliminary Chaperone-Enzyme Replacement Therapy Data Obtained in Humans
Plans to Advance Multiple Chaperone-ERT Combination Programs
FY12 Operating Expense Guidance Range of $37-$43 Million
Expects Current Cash to Fund Operations Into Mid-3Q13
Expects Current Cash to Fund Operations Into Mid-3Q13
CRANBURY, NJ, US, January 9, 2012 Amicus Therapeutics (Nasdaq: FOLD) today provided its business
outlook and financial guidance for fiscal year 2012, including details of its strategic vision and
plan. John F. Crowley, Chairman and CEO of Amicus will discuss Amicus corporate objectives and key
milestones in a presentation at the 30th Annual J.P. Morgan Healthcare Conference on
Thursday, January 12, 2012 at 8 a.m. PT (11 a.m. ET).
Mr. Crowley stated, We expect that 2012 will be a transformational year for Amicus as we look
forward to the Phase 3 data and path toward a U.S. marketing application for migalastat monotherapy
for Fabry disease. We are also greatly encouraged by the positive preliminary Chaperone-ERT data in
Fabry patients in our ongoing Phase 2 study. We believe that this combination platform technology
has the potential to improve the therapeutic efficacy and safety profile of multiple enzyme
replacement therapies for lysosomal storage diseases.
We begin 2012 as a company with late-stage clinical programs, a broad platform technology, a
strong balance sheet and a key strategic partnership with GSK. We believe that these pillars of
strength uniquely position Amicus to grow into a fully integrated biopharmaceutical company at the
forefront of developing therapies for rare and orphan diseases, concluded Mr. Crowley.
Strategic Vision and Plan
The company plans to advance in 2012 two pharmacological chaperone monotherapy programs for
genetic diseases:
| Migalastat HCl for patients with Fabry disease identified as having
alpha-galactosidase A mutations amenable to chaperone therapy |
| AT3375 for Parkinsons disease in Gaucher disease carriers and potentially the
broader Parkinsons population |
Amicus also plans to advance multiple pharmacological chaperone-ERT combination programs
for genetic diseases, including:
| Migalastat HCl co-administered with ERT for patients with Fabry disease receiving
ERT treatment, with any genetic mutation |
| AT2220 (duvoglustat HCl) co-administered with ERT for Pompe disease |
| Several new, undisclosed pharmacological chaperone programs focused on the
combination of chaperones with ERTs for additional lysosomal storage diseases (LSDs). |
Fabry Disease Programs
Migalastat HCI is an investigational oral pharmacological chaperone for the treatment of Fabry
disease being developed in collaboration with GlaxoSmithKline (GSK). Under the terms of the
collaboration, GSK has an exclusive worldwide license to develop, manufacture and commercialize
migalastat HCl.
Amicus and GSK are conducting two Phase 3 global registration studies (Study 011 and Study 012) of
migalastat HCl monotherapy, along with a Phase 2 study (Study 013) evaluating migalastat
co-adminstered with enzyme replacement therapy (ERT) for the treatment of Fabry disease.
Positive
preliminary results from the ongoing Phase 2 Study 013 were announced in a press release
last week and will be presented as a late breaking abstract at the 8th Annual Lysosomal Disease
Network WORLD Symposium (LDN WORLD) in San Diego, February 8-10, 2012.
Key objectives in 2012 for these Fabry programs include:
| Presentation of Phase 3 (Study 011) patient demographics, preliminary Phase 2
(Study 013) co-administration results, and additional abstracts at
LDN WORLD |
| Complete Phase 2 (Study 013) co-administration study in 1H12 |
| Phase 3 (Study 011) results of migalastat HCl monotherapy in 3Q12 |
| Completion of Phase 3 (Study 012) enrollment by year-end 2012 to support
additional global marketing applications |
Chaperone-ERT Combination Programs
The Company owns exclusive rights to the pharmacological chaperone AT2220 (duvoglustat HCl) for
Pompe disease. During the first half of 2012, Amicus expects to announce preliminary results from
an ongoing, open-label Phase 2 drug-drug interaction study (Study
010) of AT2220 co-administered
with the ERT alglucosidase alfa in approximately 16 individuals with Pompe disease. Study 010 is
designed to evaluate the safety of co-administered AT2220 and ERT. The study will also evaluate
the plasma pharmacokinetics of the infused enzyme with and without co-administration of AT2220, and
will assess uptake of active enzyme into skeletal muscle (quadriceps) via needle biopsy.
Data from preclinical studies in Pompe knock-out mice presented in 2011 at several scientific
symposia demonstrated that AT2220 co-administered with ERT significantly enhanced the uptake of the
active enzyme into key organs involved in Pompe disease, including heart, diaphragm, and skeletal
muscles. These preclinical data also showed a greater reduction of glycogen in key organs with the
co-administration of AT2220 versus ERT alone.
In 2012, Amicus intends to increase its commitment to the broader application of the chaperone-ERT
combination technology as a potential next-generation treatment approach for multiple
LSDs. The Company has initiated new undisclosed pharmacological chaperone research and development
programs to investigate the use of chaperones in combination with ERTs potentially to improve
treatment outcomes.
Parkinsons Disease Program
Amicus has been a pioneer in investigating the link between Gaucher and Parkinsons disease (PD),
and has been exploring the possibility of using pharmacological chaperones that target
glucocerebrosidase (GCase), the enzyme deficient in Gaucher disease, for more than five years.
In 2011, numerous peer-reviewed publications in leading scientific journals shed additional light
on the underlying mechanisms that link Gaucher and PD, and further validated GCase as a target for
the treatment of Parkinsons disease. In particular, these new papers demonstrated a direct
connection between GCase and alpha-synuclein, whose accumulation in the brain is a hallmark of PD,
and showed that increased GCase activity in the brain of mouse models could correct alpha-synuclein
pathology and other deficits.
Amicus will continue preclinical and IND-enabling studies for the pharmacological chaperone AT3375,
which targets the same GCase enzyme that is deficient in Gaucher disease. These preclinical studies
are anticipated to be complete by year-end 2012 and are funded in part by a grant awarded by the
Michael J. Fox Foundation.
FY12 Financial Guidance
The Company expects to begin 2012 with a cash balance of approximately $60 million and full-year
2012 operating expenses to total between $37 million and $43 million, net of cost sharing and
milestones related to the GSK collaboration. The Company anticipates that its current cash
position, including anticipated Fabry program reimbursements from GSK will be sufficient to fund
operations through the middle of third quarter 2013. This does not include any regulatory
milestones that the Company may be eligible to receive in this time period under its agreement with
GSK.
Amicus and GSK equally shared development costs for migalastat HCl (monotherapy and
co-administration) in 2011, and GSK is responsible for 75% of these costs in 2012 and beyond. The
Company is also eligible to receive up to $170 million in development, regulatory and commercial
milestones under the Fabry collaboration.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a biopharmaceutical company at the forefront of developing
therapies for rare and orphan diseases. The Company is developing orally-administered, small
molecule drugs called pharmacological chaperones, a novel, first-in-class approach to treating a
broad range of diseases including lysosomal storage disorders and diseases of neurodegeneration.
Amicus lead program migalastat HCl is in Phase 3 for the treatment of Fabry disease.
About Fabry Disease
Fabry disease is an inherited lysosomal storage disease that is currently estimated to affect
approximately 5,000 to 10,000 people worldwide. Fabry Disease is caused by deficiency of an enzyme
called alpha-galactosidase A (alpha-Gal A). The role of alpha-Gal A within the body is to break
down a complex lipid called globotriaosylceramide (GL-3). Reduced or absent levels of alpha-Gal A
activity leads to the accumulation of GL-3 in the affected tissues, including the central nervous
system, heart, kidneys, and skin. This accumulation of GL-3 is believed to cause the various
symptoms of Fabry disease, including pain, kidney failure, and increased risk of heart disorders
and stroke.
About Pompe Disease
Pompe disease is a lysosomal storage disease characterized by progressive skeletal muscle weakness
and respiratory insufficiency. It is caused by a deficiency in lysosomal alpha-glucosidase (GAA)
activity, which leads to accumulation of glycogen in tissues affected by the disease (primarily
muscle). Pompe disease affects an estimated 5,000 to 10,000 individuals worldwide and is clinically
heterogeneous in the age of onset, the extent of organ involvement, and the rate of progression.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private
Securities Litigation Reform Act of 1995 relating to clinical development of Amicus candidate drug
products and the timing and reporting of results from clinical trials evaluating Amicus candidate
drug products. Words such as, but not limited to, look forward to, believe, expect,
anticipate, estimate, intend, plan, targets, likely, will, would, should and
could, and similar expressions or words identify forward-looking statements. Such forward-looking
statements are based upon current expectations that involve risks, changes in circumstances,
assumptions and uncertainties. The inclusion of forward-looking statements should not be regarded
as a representation by Amicus that any of its plans will be achieved. Any or all of the
forward-looking statements in this press release may turn out to be wrong. They can be affected by
inaccurate assumptions Amicus 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 the potential goals, progress, timing and results of
clinical trials, actual results may differ materially from those set forth in this release due to
the risks and uncertainties inherent in the business of Amicus, including, without limitation: the
potential that results of clinical or pre-clinical 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 may not grant or may delay approval for our
product candidates; the potential that preclinical and clinical studies could be delayed because we
identify serious side effects or other safety issues; the potential that we will need additional
funding to complete all of our studies and, our dependence on third parties in the conduct of our
clinical studies. Further, the results of earlier preclinical studies and/or clinical trials may
not be predictive of future results. 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, 2010. 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 Amicus undertakes no obligation to revise or update this news release to
reflect events or circumstances after the date hereof. This caution is made under the safe harbor
provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.
CONTACTS:
Investors/Media:
Sara Pellegrino
spellegrino@amicustherapeutics.com
(609) 662-5044
Sara Pellegrino
spellegrino@amicustherapeutics.com
(609) 662-5044
FOLDG