Navigation Links
Amicus Therapeutics Announces Fourth Quarter and Full Year 2007 Financial Results & Program Advancements
Date:1/29/2008

- $161.5 million in cash at year end; no equity financings expected in 2008 - Significant progress in lead lysosomal disease programs to continue in 2008; latest data for all programs to be presented at ACMG meeting in March - Expansion of research and development investment in pharmacological chaperone platform planned for targets beyond lysosomal diseases in 2008

CRANBURY, N.J., Jan. 29 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD), a biopharmaceutical company developing small molecule, orally- active pharmacological chaperones for the treatment of human genetic diseases, today announced financial results for the fourth quarter and full year of 2007. On a reported basis calculated in accordance with U.S. Generally Accepted Accounting Principles (GAAP), Amicus announced a net loss attributable to common stockholders of $0.53 per share ($0.48 per share on a non-GAAP basis) for the three months ended December 31, 2007. For the year ended December 31, 2007, on a GAAP basis, the net loss attributable to common stockholders was $3.14 per share ($2.80 per share on a non-GAAP basis). As of December 31, 2007, cash, cash equivalents, and marketable securities totaled $161.5 million. Amicus Chief Financial Officer James Dentzer noted that: "We have ended 2007 in a strong financial position. We have several years of cash on hand, and this is further enhanced by the cost sharing and expected milestones from our partnership with Shire Human Genetic Therapies. As a result, we do not expect to raise cash from any equity financings in 2008."

In November 2007, Amicus entered into a strategic collaboration with Shire Human Genetic Therapies, a business unit of Shire plc, to jointly develop Amicus' three lead pharmacological41

Interest expense (73) (79) (273) (348) (1,430)

Change in fair value

of warrant liability 4 - (23) (149) (454)

Other expense - - (1,180) - (1,180)

Loss before tax

benefit (17,790) (11,773) (46,345) (41,167) (125,529)

Income tax benefit - - - - 695

Net loss (17,790) (11,773) (46,345) (41,167) (124,834)

Deemed dividend - - (19,424) - (19,424)

Preferred stock accretion (37) - (159) (351) (802)

Net loss attributable

to common

stockholders $(17,827) $ (11,773) $(65,928) $(41,518) $(145,060)

Net loss attributable

to common stockholders

per common share -

basic and diluted $ (19.77) $(0.53) $(89.58) $(3.14)

Weighted-average

common shares

outstanding - basic

and diluted 901,748 22,343,974 735,967 13,235,755

See accompanying notes to consolidated financial statements

Table 2

Amicus Therapeutics, Inc.

Reconciliation of GAAP to non-GAAP Measures for the
Statement of Operations Information for Three Months Ended December 31, 2007
(Unaudited)

(In thousands, except share and per share amounts)

Preferred

Stock Stock GAAP as

Non-GAAP Accretion Compensation Reported

Income Statement

Classifications:

Revenue $1,784 $1,784

Research and development (9,235) $- $(435) (9,670)

General and administrative (4,608) (676) (5,284)

Depreciation and amortization (313) (313)

Interest income 1,789 1,789

Interest expense (79) (79)

Summary:

Net loss: $(10,662) $- $ (1,111) $ (11,773)

Net loss per share - basic

and diluted: $(0.48) $- $(0.05) $(0.53)

Weighted average number of

shares outstanding: 22,343,974 22,343,974

Table 3

Amicus Therapeutics, Inc.

Reconciliation of GAAP to non-GAAP Measures for the
Statement of Operations Information for Three Months Ended December 31, 2006
(Unaudited)

(In thousands, except share and per share amounts)

Change

in Fair

Value

of Preferred

Warrant Stock Stock GAAP as

Non-GAAP Liability Accretion Compensation Reported

Income Statement

Classifications:

Research and

development $ (13,609) $- $- $(577) $(14,186)

General and

administrative (3,582) (448) (4,030)

Depreciation and

amortization (291) (291)

Interest income 786 786

Interest expense (73) (73)

Change in fair value of

warrant liability - 4 4

Preferred stock accretion - (37) (37)

Summary:

Net loss: $ (16,769) $4 $(37) $ (1,025) $(17,827)

Net loss per share -

basic and diluted: $(18.60) $ 0.01 $(0.04) $(1.14) $(19.77)

Weighted average

number of shares

outstanding: 901,748 901,748

Table 4

Amicus Therapeutics, Inc.

Reconciliation of GAAP to non-GAAP Measures for the

Statement of Operations Information for Year Ended December 31, 2007

(Unaudited)

(In thousands, except share and per share amounts)

Change

in Fair

Value

of Preferred

Warrant Stock Stock GAAP as

Non-GAAP Liability Accretion Compensation Reported

Income Statement

Classifications:

Revenue $1,784 $1,784

Research and

development (29,480) $- $- $(1,594) (31,074)

General and

administrative (12,887) (2,391) (15,278)

Depreciation and

amortization (1,237) (1,237)

Interest income 5,135 5,135

Interest expense (348) (348)

Change in fair value of

warrant liability - (149) (149)

Preferred stock accretion - (351) (351)

Summary:

Net loss: $(37,033) $(149) $(351) $(3,985) $(41,518)

Net loss per share -

basic and diluted: $(2.80) $(0.01) $(0.03) $(0.30) $(3.14)

Weighted average

number of shares

outstanding: 13,235,755 13,235,755

Table 5

Amicus Therapeutics, Inc.

Reconciliation of GAAP to non-GAAP Measures for the

Statement of Operations Information for Year Ended December 31, 2006

(Unaudited)

(In thousands, except share and per share amounts)

Change

in Fair

Value of Preferred

Non- Warrant Stock Deemed Stock GAAP as

GAAP Liability Accretion Dividend Compensation Reported

Income Statement

Classifications:

Research and

development $(31,924) $- $- $- $(1,706) $(33,630)

General and

administrative (10,733) (1,544) (12,277)

Depreciation and

amortization (952) (952)

Interest income 1,990 1,990

Interest expense (273) (273)

Change in fair

value of warrant

liability - (23) (23)

Other expense (1,180) (1,180)

Preferred stock

accretion - (159) (159)

Deemed dividend - (19,424) (19,424)

Summary:

Net loss: $(43,072) $(23) $(159) $(19,424) $(3,250) $(65,928)

Net loss per

share - basic

and diluted: $(58.52) $(0.03) $(0.22) $(26.39) $(4.42) $(89.58)

Weighted average

number of shares

outstanding: 735,967 735,967

Source: FOLD -G

chaperone compounds for lysosomal storage disorders. Shire licensed the rights to commercialize these products outside of the United States and Amicus retained all rights to commercialize these products in the United States.

Program Advancements:

Fabry Disease:

In December 2007, Amicus announced positive results from its Phase 2 clinical trials of Amigal(TM) for Fabry disease. 26 patients completed the study, which was designed to evaluate safety and preliminary efficacy of multiple doses and regimens of Amigal in a broad range of Fabry disease patients. A majority of patients in these studies discontinued their enzyme replacement therapy (ERT) prior to enrollment in the Amigal protocols.
Key findings include the following:

-- Amigal was generally safe and well-tolerated at all doses evaluated and

no drug-related serious adverse events were reported

-- Amigal increased the level of the enzyme deficient in Fabry patients in

24 of 26 study subjects

-- Amigal was shown to reduce the accumulated substrate in a majority of

study subjects

-- Renal and cardiac function results were encouraging, including those

seen in patients treated for nearly two years

-- Responses in patients with different Fabry mutations were consistent

with the results of in vitro testing, thus confirming the ability to

use pharmacogenetics to select likely responders for future studies

-- 23 patients have elected to continue Amigal treatment in an extension

protocol

The Phase 2 Amigal study results will be presented to the scientific and medical community at the American College of Medical Genetics (ACMG) Annual Meeting on March 12-16, 2008, in Phoenix, Arizona.

Gaucher Disease:

Amicus also recently announced positive interim results from its first Phase 2 clinical trial for Gaucher disease in patients who switched from enzyme replacement therapy (ERT) with Cerezyme(R) (imiglucerase) to the pharmacological chaperone Plicera(TM). Thirty patients were enrolled in this trial, which was designed to demonstrate safety and to evaluate various doses and dose regimens of Plicera. The study subjects on average were on ERT with Cerezyme(R) for ten years prior to entering this study.

Preliminary data are available for the first twenty study subjects. Key findings include the following:

-- Plicera was generally safe and well-tolerated at all doses evaluated

and no serious adverse events have been reported

-- The level of the enzyme deficient in people with Gaucher disease

(GCase) as measured in white blood cells increased in 15 of the 20

patients

-- In the cohort with the highest Plicera exposure, 5 out of 5 patients

responded to Plicera with, on average, a near tripling of their GCase

levels from baseline

-- The 5 patients without a clear increase in GCase levels from baseline

were in either the lowest dose cohort or the cohort dosed least

frequently

-- 16 of 20 patients in this study had at least one copy of the N370S

mutation, the most common mutation known to cause Type 1 Gaucher

disease.

"As the Gaucher program at Amicus advances, it has increasing significance and value for our company. Plicera has the potential to address the needs of the vast majority of people living with Gaucher disease and may offer a new standard of care in the treatment and maintenance of their disease. We expect to move forward aggressively in 2008 with additional studies as we explore the full potential of switching Gaucher patients from Cerezyme(R) to Plicera," said John F. Crowley, Amicus President & CEO.

Amicus expects that the results for all thirty patients enrolled in the first Plicera Phase 2 study to be presented at the ACMG meeting in March.

Pompe Disease:

During the fourth quarter of 2007, Amicus successfully completed a series of Phase I clinical studies of AT2220 in healthy volunteers. AT2220 is an orally available small-molecule pharmacological chaperone targeting the enzyme deficient in people living with Pompe disease. These studies demonstrated that AT2220 was generally safe and well-tolerated at all doses tested and that there were no drug-related serious adverse events. Amicus expects to report on these data at the ACMG conference in March along with the results of an additional clinical ex vivo response study designed to test the responsiveness of various Pompe mutations to AT2220.

"The data from these Phase 2 trials of Amigal and Plicera are part of an expanding body of scientific and clinical evidence that we are building through rigorous studies to demonstrate that many patients with Fabry, Gaucher and Pompe disease may be successfully switched from ERT to a pharmacological chaperone," noted John Crowley.

Other Protein Folding and Stabilization Programs:

In 2008, Amicus expects to accelerate its investments in research and development to better understand the potential for using pharmacological chaperones to treat a range of human genetic diseases that could benefit from improved protein folding and stabilization.

The company's advanced pre-clinical program in Parkinson's disease, funded in part by a grant from the Michael J. Fox Foundation, will continue with the potential to initiate clinical studies in 2008. In the fourth quarter of 2007, the company announced pre-clinical results which further support a biochemical link between the GCase enzyme and the accumulation of alpha- synuclein, a protein believed to play a key role in Parkinson's disease. Furthermore, in an animal model Amicus showed preliminary pre-clinical results that for the first time demonstrated that administration of a pharmacological chaperone can lead to reduction of alpha-synuclein aggregates in the brain. These results were first presented in November 2007 at the Annual Meeting for the Society for Neuroscience.

"In 2008, we plan to apply our knowledge and expertise in the fields of protein folding, genetic disease and pharmacological chaperones to the development of treatments for other human genetic diseases beyond lysosomal storage disorders. We will continue to invest heavily in our current programs while also advancing new research efforts against additional disease targets in areas such as metabolic disorders, neurodegenerative diseases and cancer," said David J. Lockhart, Amicus' Chief Scientific Officer.

Additional Financial Results & Notes

On a reported basis, the net loss attributable to common stockholders for the three months ended December 31, 2007 was $11.8 million as compared to $17.8 million for the same period in 2006. On a non-GAAP basis, the net loss for the three months ended December 31, 2007 was $10.7 million as compared to $16.8 million and the same period in 2006.

Amicus recorded revenue during the fourth quarter of 2007 that represents two different revenue streams from the Shire agreement. Upon signing the agreement, Amicus received an upfront payment of $50 million that will be recognized as revenue on a straight-line basis over 18 years from the date of the agreement. The upfront payment for the period of November 7 to December 31, or $0.4 million, was recognized in the fourth quarter of 2007. The remainder of the upfront payment was recorded as deferred revenue. Additionally, Amicus recognized $1.4 million of Research Revenue on reimbursed research and developments costs for the period of November 7 to December 31, in the fourth quarter of 2007.

The differences between U.S. GAAP and non U.S. GAAP financial results are itemized in tables 2 through 5, and are primarily due to:

-- Pre-tax stock compensation expense

-- Pre-tax charges for preferred stock accretion

-- Pre-tax charges for changes in the fair value of warrant liability

-- Deemed dividend

Use of Non-GAAP Financial Measures

Amicus' "non-GAAP net loss" and "non-GAAP diluted net loss per common share" financial measures are defined as reported, or GAAP net loss and diluted net loss per common share excluding certain items further discussed below. Amicus' management uses these non-GAAP financial measures to establish financial goals and to gain an understanding of the comparative financial performance of Amicus from year to year and quarter to quarter. Accordingly, Amicus believes investors' understanding of Amicus' financial performance is enhanced as a result of disclosing these non-GAAP financial measures. Non-GAAP net loss and diluted net loss per common share should not be viewed in isolation or as a substitute for reported, or GAAP net loss and diluted net loss per common share.

(1) Stock option expense - Non-GAAP net loss and diluted net loss per

common share exclude the impact of the stock option expense recorded

in accordance with SFAS No. 123R. Amicus believes that excluding the

impact of expensing stock options better reflects the recurring

economic characteristics of its business.

(2) Other items - Non-GAAP net loss and diluted net loss per common share

exclude other unusual or non-recurring items that are evaluated on an

individual basis. Amicus' evaluation of whether to exclude an item for

purposes of determining its non-GAAP financial measures considers both

the quantitative and qualitative aspects of the item, including, among

other things (i) its size and nature, (ii) whether or not it relates

to its ongoing business operations, and (iii) whether or not Amicus

expects it to occur as part of its normal business on a regular basis.

Items excluded for purposes of determining non-GAAP net loss and

diluted net loss per common share include deemed dividends, preferred

stock accretion, and changes in the fair value of warrant liability.

About Amicus Therapeutics

Amicus Therapeutics is a biopharmaceutical company developing novel, oral therapeutics known as pharmacological chaperones for the treatment of a range of human genetic diseases. Pharmacological chaperone technology involves the use of small molecules that selectively bind to and stabilize proteins in cells, leading to improved protein folding and trafficking, and increased activity. Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genetic diseases with unmet medical needs. Amicus has completed Phase 2 clinical trials of Amigal(TM) for the treatment of Fabry disease and is conducting Phase 2 clinical trials of Plicera(TM) for the treatment of Gaucher disease. The Company recently completed Phase I clinical trials of AT2220 for the treatment of Pompe disease.

Forward-Looking Statements

Amicus cautions you that statements included in this press release that are not a description of historical facts are "forward-looking statements" within the meaning of Section 21E of the Private Securities Litigation Reform Act of 1995. 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 potential progress 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 effect of the completion of the Phase 2 clinical trial for Amigal(TM) for the treatment of Fabry disease, the plans for the Phase 3 clinical trial for Amigal(TM), the Phase 2 clinical trials for Plicera(TM) for the treatment of Gaucher disease and the effect of the completion of the Phase 1 clinical trials for AT2220 for the treatment of Pompe disease. In addition, the amount and impact of stock- based compensation charges, royalty fees, and pre-tax charges for preferred stock accretion and warrant liability and Amicus' definition of "non-GAAP net income" and/or "non-GAAP net income per share" do not constitute guarantees of future performance and are subject to a variety of risks and uncertainties that could cause its actual results to differ materially from those anticipated. Further, the results of earlier clinical trials may not be predictive of future results; Amicus and its licensors may not be able to obtain, maintain and successfully enforce adequate patent and other intellectual property protection of its product candidates; and other risks detailed in the public filings of Amicus 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.

FOLD -G

Table 1

Amicus Therapeutics, Inc.

(a development stage company)

Consolidated Statements of Operations

(Unaudited)

(In thousands, except share and per share amounts)

Period

from

February

4, 2002

inception

to

Three Months Twelve Months December

Ended December 31, Ended December 31, 31,

2006 2007 2006 2007 2007

Revenue:

Research revenue $- $1,375 $- $1,375 $1,375

Collaboration revenue - 409 - 409 409

Total revenue - 1,784 - 1,784 1,784

Operating Expenses:

Research and

development 14,186 9,670 33,630 31,074 89,878

General and

administrative 4,030 5,284 12,277 15,278 38,070

Impairment of

leasehold improvements - - - - 1,030

Depreciation and

amortization 291 313 952 1,237 2,794

In-process research

and development - - - - 418

Total operating

expenses 18,507 15,267 46,859 47,589 132,190

Loss from operations (18,507) (13,483) (46,859) (45,805) (130,406)

Other income (expenses):

Interest income 786 1,789 1,990 5,135 7,9
'/>"/>

SOURCE Amicus Therapeutics
Copyright©2008 PR Newswire.
All rights reserved

Related biology technology :

1. Amicus Therapeutics Announces Positive Results From Phase 2 Clinical Trials of Amigal(TM) for Fabry Disease
2. Amicus Therapeutics to Host R&D Day
3. Amicus Therapeutics to Present at the Lazard Capital Markets Fourth Annual Healthcare Conference
4. Meredith Martin Addy Named Chair of AIPLAs Amicus Committee
5. Amicus Therapeutics Announces Third Quarter 2007 Financial Results
6. Amicus Therapeutics to Present at the Merrill Lynch Global Pharmaceutical, Biotech & Medtech 2007 Conference
7. Allos Therapeutics to Present at the Merrill Lynch Global Pharmaceutical, Biotechnology and Medical Device Conference
8. Cell Therapeutics, Inc. (CTI) Shareholders Authorize Share Increase at Special Meeting of Shareholders
9. Velcura Therapeutics, Inc. Can Begin Clinical Trials Following Successful Investigational New Drug Application to U.S. FDA
10. Nile Therapeutics Appoints Gregory W. Schafer to Board of Directors
11. Arno Therapeutics Completes License Agreements for the Exclusive Rights to Novel, Targeted Cancer Compounds
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:2/11/2016)... , Feb. 11, 2016   BioInformant ... report, "Stem Cell Research Products, Opportunities, Tools, and Technologies ... ... in the stem cell industry, BioInformant has more than ... the stem cell market, by stem cell type. This ...
(Date:2/11/2016)... ... February 11, 2016 , ... Global ... new agreement with Bankok,Thailand-based Global Stem Cells Network (GSCN) to distribute exosome injection ... American countries, including Mexico, Costa Rica, Dominican Republic, Colombia, Argentina, Nicaragua, Panama, El ...
(Date:2/10/2016)... /PRNewswire/ - BioAmber Inc. (NYSE: BIOA ), a ... Mitsui & Co. Ltd., its partner in the ... investing an additional CDN$25 million in the joint venture ... 30% to 40%.  Mitsui will also play a stronger ... Sarnia , providing dedicated resources alongside ...
(Date:2/10/2016)... -- The Maryland House of Delegates and House Speaker ... Maryland School of Medicine Dean E. Albert Reece ... System President and CEO Robert Chrencik , MBA, ... given to the public by the leader of the ... and Mr. Chrencik for their contributions to our statewide ...
Breaking Biology Technology:
(Date:2/2/2016)... 2, 2016   Parabon NanoLabs (Parabon) ... Army Research Office and the Defense Forensics and ... of the company,s Snapshot Kinship Inference ... more generally, defense-related DNA forensics.  Although Snapshot is ... appearance and ancestry from DNA evidence), it also ...
(Date:2/1/2016)...  Today, the first day of American Heart Month, ... a first of its kind workplace health solution that ... the first application of Watson ... and Welltok will create a new offering that combines ... delivered on Welltok,s health optimization platform. The effort is ...
(Date:1/27/2016)... CHESTER, Ohio , Jan. 27, 2016  Rite ... supplier based in West Chester, Ohio ... their award winning service staff, based in ... technical capacity and ability to provide modifications, installations and ... John Dovalina , CEO of PLUS, commented, "PLUS has ...
Breaking Biology News(10 mins):