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8-K - FORM 8-K - AMICUS THERAPEUTICS, INC. | c94539e8vk.htm |
EX-99.1 - EXHIBIT 99.1 - AMICUS THERAPEUTICS, INC. | c94539exv99w1.htm |
Exhibit 99.2
Amicus Therapeutics Outlines 2010 Strategic Priorities and Continued Progress with Product Pipeline
Company to focus on advancing AmigalTM Phase 3 Program in Fabry disease
Cranbury, NJ, January 11, 2010 Amicus Therapeutics (NASDAQ: FOLD) today outlined the Companys
three key strategic priorities and presented a corporate outlook for 2010 at the 28th
Annual J.P. Morgan Healthcare Conference.
In 2010 our strategic priorities are clear and we remain steadfast in executing our business
goals. We are committed to focusing our resources on three value-creating centers within Amicus.
Advancing our global Amigal Phase 3 program is our number one priority. We expect to make
significant progress with Amigal by fully enrolling our 011 study and expect to commence the 012
Phase 3 study to support approval in the EU. We plan to balance the execution of this global,
late-stage clinical program in Fabry disease with significant progress in our chaperone-ERT
combination programs and our preclinical programs in Parkinsons disease and Alzheimers disease,
said John F. Crowley, Amicus President and CEO.
Amigal (migalastat hydrochloride) for the treatment of Fabry Disease
The Phase 3 study intended to support approval in the United States (Study 011) commenced in the
second quarter of 2009 and treatment of the first patient began in the fourth quarter of 2009. The
Company expects to complete enrollment by the end of 2010 and to have results from this study in
mid-2011. The 011 study is a 6-month, randomized, double-blind trial comparing Amigal to placebo
in approximately 60 subjects. The surrogate primary endpoint is the change in the amount of kidney
interstitial capillary GL-3. Subjects to be enrolled are Fabry patients who have never received
enzyme replacement therapy (ERT), or who have not received ERT for at least 6 months, and who have
a mutation responsive to Amigal. The Company intends to seek Accelerated Approval for Amigal
according to Subpart H regulations. The key elements of this study design and regulatory path were
agreed to with the U.S. Food and Drug Administration (FDA) in the second quarter of 2009.
Amicus previously reported that it completed a series of discussions with the European Medicines
Agency (EMEA) regarding the clinical study required for Amigal registration in Europe. The Company
today reported that it expects to commence this Phase 3 trial (Study 012) before year end. The 012
study will be an 18-month, randomized, open-label study comparing Amigal versus ERT in
approximately 60 subjects. The primary endpoint will be renal function as measured by glomerular
filtration rate (GFR). Subjects to be enrolled will be Fabry patients who are receiving ERT and
have a mutation responsive to Amigal. Patients will be randomized to switch to Amigal or to
continue receiving ERT.
Twenty-two of the 26 subjects enrolled in the original Phase 2 studies continue to receive
treatment in an ongoing extension study designed to evaluate the long-term safety and efficacy of
Amigal. Fourteen subjects have been on treatment for at least 2 years and eight subjects have been
on treatment for more than 3 years. Preliminary data from this study suggest that Amigal continues
to be generally well-tolerated. In addition, in the subset of these patients that meet the Phase 3
study entry criteria and have only received the Phase 3 dose and regimen, results suggest
stabilization of renal function as measured by GFR and trends of improvement in proteinuria. The
Company will provide an update from this study in the first quarter of 2010.
Chaperone-ERT Combination Therapy
Amicus continues to advance its program evaluating the use of pharmacological chaperones in
combination with ERT as an expansion of the chaperone technology platform. Amicus previously
reported preclinical data at several scientific conferences in 2009 demonstrating that the addition
of a pharmacological chaperone to ERT has the potential to address key limitations of ERT. The
addition of a pharmacological chaperone has been shown to prevent the loss of activity of ERT in
the circulation, increase tissue uptake and substrate reduction, and reduce antibody response.
Preclinical proof-of-concept has been established for Fabry disease and Pompe disease and initial
proof-of-concept work is currently being conducted for Gaucher disease.
The Company has selected Amigal as its first clinical candidate and plans to initiate a Phase 2
study with Amigal in combination with ERT before the end of 2010. Additionally, the Company is
evaluating options to advance Chaperone-ERT combination therapy programs for Pompe disease and
Gaucher disease.
The Company expects to report additional proof-of-concept data in Fabry, Pompe and Gaucher at
various scientific conferences throughout 2010.
Diseases of Neurodegeneration
Amicus is committed to advancing its pharmacological chaperone technology for the treatment of
diseases of neurodegeneration. The Company has advanced its preclinical program in Parkinsons
disease and established initial proof-of-concept in animal models of the disease. The Company
previously reported data demonstrating the prevention of synuclein accumulation in the brain after
treatment with the chaperone. Today the Company announced that in recent preclinical studies
treatment with a chaperone resulted in encouraging improvements in behavioral characteristics and
motor function in Parkinsons animal models.
The Company expects to complete advanced preclinical proof-of-concept studies in Parkinsons
disease during the course of the year and plans to report additional data at a scientific
conference in the second half of 2010.
Additionally, Amicus announced today that its second lead preclinical program using the
pharmacological chaperone approach is for the treatment of Alzheimers disease. The Company
expects to complete initial proof-of-concept studies during 2010 and report data in the second half
of 2010.
2010 Financial Guidance
Amicus expects to begin 2010 with a cash balance of approximately $78
million. The Company expects to spend a total of $40 to $50 million on 2010 operating expenses.
The current cash position is expected to be sufficient to fund operations and capital expenditure
requirements into the second half of 2011. In 2010, the Company plans to evaluate all opportunities
to continue to build on its financial strength including a range of potential global partnerships.
About Amicus Therapeutics
Amicus Therapeutics 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 is in Phase 3
for the treatment of Fabry disease.
Forward-Looking Statements
This 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
Amicus candidate drug products, the timing and reporting of results from preclinical studies and
clinical trial evaluating Amicus candidate drug products, and the projected cash position for the
Company. 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 ongoing discussions
with regulatory authorities and 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 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 preclinical and clinical studies could be
delayed because we identify serious side effects or other safety issues; 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. Additionally, with respect
to statements regarding projections of the Companys cash position, actual results may differ based
on market factors and the Companys ability to execute its operational and budget plans.
Additionally, all forward looking statements are subject to other risks detailed in our Annual
Report on Form 10-K for the year ended December 31, 2008, and our other public filings with the
Securities and Exchange Commission. 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:
Jenene Thomas
Director, Investor Relations
(609) 662-5084
Jenene Thomas
Director, Investor Relations
(609) 662-5084
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