10-Q
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 10-Q
(Mark One)
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þ |
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QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
For the quarterly period ended September 30, 2009
OR
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o |
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TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
For the transition period from to
Commission file number 001-33497
Amicus Therapeutics, Inc.
(Exact Name of Registrant as Specified in Its Charter)
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Delaware
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71-0869350 |
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(State or Other Jurisdiction of
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(I.R.S. Employer |
Incorporation or Organization)
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Identification Number) |
6 Cedar Brook Drive, Cranbury, NJ 08512
(Address of Principal Executive Offices and Zip Code)
Registrants Telephone Number, Including Area Code: (609) 662-2000
Indicate by check mark whether the registrant (1) has filed all reports required to be filed
by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or
for such shorter period that the registrant was required to file such reports), and (2) has been
subject to such filing requirements for the past 90 days: Yes þ No o
Indicate by check mark whether the registrant has submitted electronically and
posted on its corporate Web site, if any, every Interactive Data File required to be submitted and
posted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12
months (or for such shorter period that the registrant was required to submit and post such files).
Yes o No o
Indicate by check mark whether the registrant is a large accelerated filer, an
accelerated filer, a non-accelerated filer or a smaller-reporting company. See definition of large
accelerated filer, accelerated filer and smaller-reporting company in Rule 12b-2 of the
Exchange Act. (Check one):
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Large accelerated filer o
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Accelerated filer o
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Non-accelerated filer þ
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Smaller Reporting Company o |
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(Do not check if a smaller reporting company)
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Indicate by check mark whether the registrant is a shell company (as defined in Rule
12b-2 of the Exchange Act): Yes o No þ
The number of shares outstanding of the registrants common stock, $.01 par value per
share, as of October 28, 2009 was 22,647,869 shares.
AMICUS THERAPEUTICS, INC
Form 10-Q for the Quarterly Period Ended September 30, 2009
We have filed applications to register certain trademarks in the United States and abroad,
including AMICUSTM, AMICUS THERAPEUTICSTM (and design), AMIGALTM
and PLICERATM.
-2-
SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
This quarterly report on Form 10-Q contains forward-looking statements that involve
substantial risks and uncertainties. All statements, other than statements of historical facts,
included in this quarterly report on Form 10-Q regarding our strategy, future operations, future
financial position, future revenues, projected costs, prospects, plans and objectives of management
are forward-looking statements. The words anticipate, believe, estimate, expect, intend,
may, plan, predict, project, will, would and similar expressions are intended to
identify forward-looking statements, although not all forward-looking statements contain these
identifying words.
The forward-looking statements in this quarterly report on Form 10-Q include, among other
things, statements about:
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our plans to develop, seek regulatory approval for and commercialize Amigal, Plicera and
AT2220; |
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our ongoing and planned discovery programs, preclinical studies and clinical trials; |
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our ability to enter into selective collaboration arrangements and obtain milestone,
royalty or other payments from any such collaborators; |
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the timing of our initiation of the Phase 3 clinical study required for Amigal
registration in Europe; |
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the timing of and our ability to obtain and maintain regulatory approvals for our
product candidates; |
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the rate and degree of market acceptance and clinical utility of our products; |
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our ability to quickly and efficiently identify and develop product candidates; |
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the extent to which our scientific approach may potentially address a broad range of
diseases across multiple therapeutic areas; |
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our commercialization, marketing and manufacturing capabilities and strategy; |
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our ability to execute our operational and business plans; |
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our intellectual property position; |
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our estimates regarding expenses, future revenues, capital requirements and needs for
additional financing; and |
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our belief about our ability to fund our operating expenses. |
We may not actually achieve the plans, intentions or expectations disclosed in our
forward-looking statements, and you should not place undue reliance on our forward-looking
statements. Actual results or events could differ materially from the plans, intentions and
expectations disclosed in the forward-looking statements we make. We have included important
factors in the cautionary statements included in Part I Item 1A Risk Factors of this
Quarterly Report on Form 10-Q for the quarter ended September 30, 2009 that we believe could cause
actual results or events to differ materially from the forward-looking statements that we make.
Our forward-looking statements do not reflect the potential impact of any future acquisitions,
mergers, dispositions, joint ventures, collaborations or investments we may make.
You should read this quarterly report on Form 10-Q in conjunction with the documents that we
reference herein. We do not assume any obligation to update any forward-looking statements.
-3-
PART I. FINANCIAL INFORMATION
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Item 1. |
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Financial Statements (unaudited) |
Amicus Therapeutics, Inc.
(a development stage company)
Consolidated Balance Sheets
(Unaudited)
(in thousands, except share and per share amounts)
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December 31, |
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September 30, |
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2008 |
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2009 |
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Assets: |
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Current assets: |
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Cash and cash equivalents |
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$ |
28,073 |
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$ |
20,487 |
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Investments in marketable securities |
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93,051 |
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68,784 |
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Prepaid expenses and other current assets |
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2,463 |
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4,305 |
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Total current assets |
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123,587 |
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93,576 |
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Property and equipment, less accumulated depreciation and amortization of
$4,260 and $5,832 at December 31, 2008 and September 30, 2009,
respectively |
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4,919 |
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5,049 |
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Other non-current assets |
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267 |
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267 |
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Total Assets |
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$ |
128,773 |
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$ |
98,892 |
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Liabilities and Stockholders Equity |
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Current liabilities: |
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Accounts payable and accrued expenses |
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$ |
8,796 |
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$ |
12,759 |
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Current portion of deferred revenue |
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3,705 |
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2,778 |
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Current portion of capital lease obligations |
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877 |
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397 |
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Current portion of secured loan |
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1,148 |
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Total current liabilities |
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13,378 |
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17,082 |
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Deferred revenue, less current portion |
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44,035 |
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41,952 |
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Capital lease obligations, less current portion |
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317 |
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86 |
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Secured loan, less current portion |
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2,610 |
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Commitments and contingencies |
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Stockholders equity: |
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Common stock, $.01 par value, 50,000,000 shares authorized,
22,634,711 shares
issued and outstanding at December 31, 2008, 50,000,000 shares
authorized, 22,647,869 shares issued and outstanding at
September 30, 2009 |
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287 |
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287 |
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Additional paid-in capital |
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234,412 |
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240,499 |
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Accumulated other comprehensive income |
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533 |
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88 |
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Deficit accumulated during the development stage |
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(164,189 |
) |
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(203,712 |
) |
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Total stockholders equity |
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71,043 |
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37,162 |
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Total Liabilities and Stockholders Equity |
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$ |
128,773 |
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$ |
98,892 |
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See accompanying notes to consolidated financial statements
-4-
Amicus Therapeutics, Inc.
(a development stage company)
Consolidated Statements of Operations
(Unaudited)
(in thousands, except share and per share amounts)
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Period from |
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February 4, |
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2002 |
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(inception) |
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to |
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Three Months |
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Nine Months |
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September |
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Ended September 30, |
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Ended September 30, |
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30, |
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2008 |
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2009 |
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2008 |
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2009 |
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2009 |
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Revenue: |
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Research revenue |
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$ |
2,959 |
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$ |
4,219 |
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$ |
8,539 |
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$ |
12,799 |
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$ |
26,362 |
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Collaboration revenue |
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694 |
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694 |
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2,083 |
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2,083 |
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5,270 |
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Total revenue |
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$ |
3,653 |
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$ |
4,913 |
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$ |
10,622 |
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$ |
14,882 |
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$ |
31,632 |
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Operating Expenses: |
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Research and development |
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$ |
8,200 |
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$ |
12,609 |
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$ |
23,989 |
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$ |
37,954 |
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$ |
165,596 |
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General and
administrative |
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4,371 |
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5,217 |
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14,676 |
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15,635 |
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73,371 |
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Impairment of leasehold
improvements |
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1,030 |
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Depreciation and
amortization |
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382 |
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561 |
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1,036 |
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1,585 |
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5,872 |
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In-process research and
development |
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418 |
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Total operating expenses |
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12,953 |
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18,387 |
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39,701 |
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55,174 |
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246,287 |
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Loss from operations |
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(9,300 |
) |
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(13,474 |
) |
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(29,079 |
) |
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(40,292 |
) |
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(214,655 |
) |
Other income (expenses): |
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Interest income |
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1,019 |
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129 |
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4,053 |
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924 |
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13,684 |
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Interest expense |
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(49 |
) |
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(84 |
) |
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(179 |
) |
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(155 |
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(1,802 |
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Change in fair value of
warrant liability |
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(454 |
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Other expense |
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(1,180 |
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Loss before tax benefit |
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(8,330 |
) |
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(13,429 |
) |
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(25,205 |
) |
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(39,523 |
) |
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(204,407 |
) |
Benefit from income taxes |
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150 |
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695 |
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Net loss |
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(8,180 |
) |
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(13,429 |
) |
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(25,205 |
) |
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(39,523 |
) |
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(203,712 |
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Deemed dividend |
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(19,424 |
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Preferred stock accretion |
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(802 |
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Net loss attributable to
common stockholders |
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$ |
(8,180 |
) |
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$ |
(13,429 |
) |
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$ |
(25,205 |
) |
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$ |
(39,523 |
) |
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$ |
(223,938 |
) |
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Net loss attributable to
common stockholders per
common share basic and
diluted |
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$ |
(0.36 |
) |
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$ |
(0.59 |
) |
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$ |
(1.12 |
) |
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$ |
(1.75 |
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Weighted-average common shares
outstanding
basic and diluted |
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22,517,431 |
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22,621,513 |
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22,465,981 |
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22,617,808 |
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See accompanying notes to consolidated financial statements
-5-
Amicus Therapeutics, Inc.
(a development stage company)
Consolidated Statements of Cash Flows
(Unaudited)
(in thousands)
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Period from |
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February 4, 2002 |
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Nine Months |
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(inception) to |
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Ended September 30, |
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September 30, |
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2008 |
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2009 |
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2009 |
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Operating activities |
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Net loss |
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$ |
(25,205 |
) |
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$ |
(39,523 |
) |
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$ |
(203,712 |
) |
Adjustments to reconcile net loss to net cash used
in operating activities: |
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Non-cash interest expense |
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|
525 |
|
Depreciation and amortization |
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1,036 |
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1,585 |
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5,870 |
|
Amortization of non-cash compensation |
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522 |
|
Stock-based compensation employees |
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4,819 |
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6,039 |
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19,125 |
|
Stock-based compensation non-employees |
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|
853 |
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Stock-based license payments |
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1,220 |
|
Change in fair value of warrant liability |
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|
454 |
|
Loss on disposal of asset |
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9 |
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54 |
|
Impairment of leasehold improvements |
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1,030 |
|
Non-cash charge for in-process research and development |
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|
418 |
|
Beneficial conversion feature related to bridge financing |
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|
135 |
|
Changes in operating assets and liabilities: |
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Prepaid expenses and other current assets |
|
|
(477 |
) |
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(1,842 |
) |
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(4,305 |
) |
Other non-current assets |
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|
(236 |
) |
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(289 |
) |
Accounts payable and accrued expenses |
|
|
(661 |
) |
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|
3,963 |
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|
12,759 |
|
Deferred revenue |
|
|
(1,841 |
) |
|
|
(3,010 |
) |
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|
44,730 |
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Net cash used in operating activities |
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(22,565 |
) |
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(32,779 |
) |
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(120,611 |
) |
Investing activities |
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Sale and redemption of marketable securities |
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125,925 |
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106,455 |
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453,622 |
|
Purchases of marketable securities |
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(125,326 |
) |
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(82,634 |
) |
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(522,435 |
) |
Purchases of property and equipment |
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(1,663 |
) |
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(1,724 |
) |
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(12,003 |
) |
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Net cash (used in)/provided by investing activities |
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(1,064 |
) |
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22,097 |
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(80,816 |
) |
Financing activities |
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Proceeds from the issuance of preferred stock,
net of issuance costs |
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143,022 |
|
Proceeds from the issuance of common stock, net of issuance costs |
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68,093 |
|
Proceeds from the issuance of convertible notes |
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5,000 |
|
Payments of capital lease obligations |
|
|
(1,148 |
) |
|
|
(710 |
) |
|
|
(5,103 |
) |
Proceeds from exercise of stock options |
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|
444 |
|
|
|
48 |
|
|
|
1,269 |
|
Proceeds from exercise of warrants (common and preferred) |
|
|
|
|
|
|
|
|
|
|
264 |
|
Proceeds from capital asset financing arrangement |
|
|
|
|
|
|
|
|
|
|
5,611 |
|
Proceeds from secured loan arrangement |
|
|
|
|
|
|
3,758 |
|
|
|
3,758 |
|
|
|
|
|
|
|
|
|
|
|
Net cash (used in)/ provided by financing activities |
|
|
(704 |
) |
|
|
3,096 |
|
|
|
221,914 |
|
|
|
|
|
|
|
|
|
|
|
Net (decrease)/ increase in cash and cash equivalents |
|
|
(24,333 |
) |
|
|
(7,586 |
) |
|
|
20,487 |
|
Cash and cash equivalents at beginning of period |
|
|
44,188 |
|
|
|
28,073 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cash and cash equivalents at end of period |
|
$ |
19,855 |
|
|
$ |
20,487 |
|
|
$ |
20,487 |
|
|
|
|
|
|
|
|
|
|
|
Supplemental disclosures of cash flow information |
|
|
|
|
|
|
|
|
|
|
|
|
Cash paid during the period for interest |
|
$ |
179 |
|
|
$ |
155 |
|
|
$ |
1,509 |
|
Non-cash activities |
|
|
|
|
|
|
|
|
|
|
|
|
Conversion of notes payable to preferred stock |
|
$ |
|
|
|
$ |
|
|
|
$ |
5,000 |
|
Conversion of preferred stock to common stock |
|
$ |
|
|
|
$ |
|
|
|
$ |
148,591 |
|
Accretion of redeemable convertible preferred stock |
|
$ |
|
|
|
$ |
|
|
|
$ |
802 |
|
Beneficial conversion feature related to the issuance |
|
|
|
|
|
|
|
|
|
|
|
|
of Series C redeemable convertible preferred stock |
|
$ |
|
|
|
$ |
|
|
|
$ |
19,424 |
|
See accompanying notes to consolidated financial statements
-6-
Note 1. Description of Business and Significant Accounting Policies
Corporate Information, Status of Operations and Management Plans
Amicus Therapeutics, Inc. (the Company) was incorporated on February 4, 2002 in Delaware for
the purpose of creating a premier drug development company at the forefront of therapy for human
genetic diseases initially based on intellectual property in-licensed from Mount Sinai School of
Medicine. The Companys activities since inception have consisted principally of raising capital,
establishing facilities, and performing research and development, including clinical trials.
Accordingly, the Company is considered to be in the development stage.
In November 2007, the Company entered into a License and Collaboration Agreement with
Shire Pharmaceuticals Ireland Ltd. (Shire). Under the agreement, the Company and Shire were
jointly developing the Companys three lead pharmacological chaperone compounds for lysosomal
storage disorders: Amigal (migalastat hydrochloride), Plicera (afegostat tartrate) and AT2220
(1-deoxynojirimycin HCl). On October 29, 2009, the Company and Shire agreed to mutually
terminate the agreement. As a result of this termination, Amicus has reacquired all global
development and commercialization rights from Shire for these lead
programs, and received
a $5.2 million payment from Shire as full and final payment for amounts due to
the Company under the collaboration. For further information, see Note 7.
Development and Commercialization Agreement with Shire.
The Company has an accumulated deficit of approximately $203.7 million at September 30, 2009
and anticipates incurring losses through the year 2009 and beyond. The Company has not yet
generated commercial sales revenues and has been able to fund its operating losses to date through
the sale of its redeemable convertible preferred stock, issuance of convertible notes, net proceeds
from our initial public offering (IPO), the upfront licensing payment from Shire and other
financing arrangements. The Company believes that its existing cash and cash equivalents and
short-term investments will be sufficient to cover its cash flow requirements for 2009.
Basis of Presentation
The Company has prepared the accompanying unaudited consolidated financial statements in
accordance with accounting principles generally accepted in the United States of America (U.S.
GAAP) for interim financial information and with the instructions to Form 10-Q and Article 10-01 of
Regulations S-X. Accordingly, they do not include all of the information and disclosures required
by generally accepted accounting principles for complete financial statements. In the opinion of
management, the accompanying unaudited financial statements reflect all adjustments, which include
only normal recurring adjustments, necessary to present fairly the Companys interim financial
information.
The accompanying unaudited consolidated financial statements and related notes should be read
in conjunction with the Companys financial statements and related notes as contained in the
Companys Annual Report on Form 10-K for the year ended December 31, 2008. For a complete
description of the Companys accounting policies, please refer to the Annual Report on Form 10-K
for the fiscal year ended December 31, 2008.
Revenue Recognition
The Company recognizes revenue when amounts are realized or realizable and earned. Revenue is
considered realizable and earned when the following criteria are met: (1) persuasive evidence of an
arrangement exists; (2) delivery has occurred or services have been rendered; (3) the price is
fixed or determinable; and (4) collection of the amounts due are reasonably assured.
In determining the accounting for collaboration agreements, the Company determines whether an
arrangement involves multiple revenue-generating deliverables that should be accounted for as a
single unit of accounting or divided into separate units of accounting for revenue recognition
purposes. If this division is required, the arrangement consideration should be allocated among
the separate units of accounting. If the arrangement represents a single unit of accounting, the
revenue recognition policy and the performance obligation period must be determined (if not already
contractually defined) for the entire arrangement. If the arrangement represents separate
units of accounting according to the separation criteria, a revenue recognition policy must be
determined for each unit. Revenues for non-refundable upfront license fee payments will be
recognized on a straight line basis as Collaboration Revenue over the period of the performance
obligations.
-7-
The revenue associated with reimbursements for research and development costs under
collaboration agreements is included in Research Revenue and the costs associated with these
reimbursable amounts are included in research and development expenses. The Company records these
reimbursements as revenue and not as a reduction of research and development expenses as the
Company has the risks and rewards as the principal in the research and development activities.
Income Taxes
The Company accounts for income taxes under the liability method. Under this method deferred
income tax liabilities and assets are determined based on the difference between the financial
statement carrying amounts and tax basis of assets and liabilities and for operating losses and tax
credit carryforwards, using enacted tax rates in effect in the years in which the differences are
expected to reverse. A valuation allowance is recorded if it is more likely than not that a
portion or all of a deferred tax asset will not be realized.
New Accounting Standards
In October 2009, the Financial Accounting Standards Board (FASB) issued guidance on
revenue recognition related to multiple-element arrangements. This new guidance requires
companies to allocate revenue in multiple-element arrangements based on an elements estimated
selling price if vendor-specific or other third party evidence of value is not available. This
guidance is effective prospectively for revenue arrangements entered into or materially
modified in fiscal years beginning on or after June 15, 2010. Early adoption is permitted
retrospectively from the beginning of an entitys fiscal year. The Company does not expect
this will have a significant impact on the financial statements of the Company.
In June 2009, the FASB issued The FASB Accounting Standards Codification (the
Codification), which became the source of U.S. generally accepted accounting principles to be
applied to nongovernmental entities. The Codification superseded all existing non-SEC
accounting and reporting standards and was effective for financial statements issued for
interim and annual periods ending after September 15, 2009. Since it is not intended to change
or alter existing U.S. GAAP, this pronouncement did not have any impact on the Companys
financial statements.
In June 2009, the FASB issued guidance on the accounting for and disclosure of subsequent
events. This guidance required application of the requirements to interim or annual financial
periods ending after June 15, 2009. The adoption of this pronouncement did not have a material
effect on the financial statements of the Company.
At its April 2009 Board meeting, the FASB issued guidance related to the reporting of
financial instruments which included the following:
|
|
|
Guidance on the recognition of an Other Than Temporary Impairment and new
disclosure requirements. The recognition and presentation provisions apply only to
debt securities classified as available for sale and held to maturity. |
|
|
|
Extension of the fair value disclosure requirements of the fair value of all
financial instruments (recognized or unrecognized) to interim financial statements
of publicly traded companies, when practicable to do so. These fair value
disclosures must be presented together with the carrying amount of the financial
instruments in a manner that clearly distinguishes between assets and liabilities
and indicates how the carrying amounts relate to amounts reported on the balance
sheet. An entity must also disclose the methods and significant assumptions used
to estimate the fair value of the financial instruments. |
|
|
|
Guidance on estimating fair value when the volume and level of activity for an
asset or liability has significantly decreased in relation to normal market
activity for the asset or liability. |
-8-
The guidance listed above was effective for interim and annual periods ending after June
15, 2009. The adoption of these pronouncements did not have a material effect on the
financial statements of the Company and the additional disclosures required are included in
the financial statements of the Company for the period ended September 30, 2009.
Subsequent Events
The Company evaluated events that occurred subsequent to September 30, 2009 through the date
of issuance of these financial statements on November 4, 2009. The following events are noted:
|
|
|
On October 29, 2009, the Company announced the mutual termination of the license and
collaboration agreement with Shire as discussed in Note 7. Development and
Commercialization Agreement with Shire. |
|
|
|
On October 29, 2009, the Company also announced a workforce reduction of 26
employees, or approximately 20 percent of its workforce, as part of a corporate
restructuring designed to reduce costs and align the Companys resources with its key
strategic priorities. The Company generally expects to complete the workforce
reduction by the end of the fourth quarter of 2009. The Company estimates that it will
record charges of approximately $0.9 million during the fourth quarter of 2009 for
employment termination costs payable in cash in connection with the workforce
reduction. |
|
|
|
On November 4, 2009, as a result of the termination
discussed above, the Company received a $5.2 million payment
from Shire as full and final payment for the amounts due to the
Company. |
Except for these items noted above, there were no material recognized or non-recognized
subsequent events during this period.
Note 2. Cash and Available for Sale Investments
As of September 30, 2009, the Company held $20.5 million in cash and cash equivalents and
$68.8 million of available for sale investment securities which are reported at fair value on the
Companys balance sheet. Unrealized holding gains and losses are reported within accumulated other
comprehensive income/(loss) as a separate component of stockholders equity. If a decline in the
fair value of a marketable security below the Companys cost basis is determined to be other than
temporary, such marketable security is written down to its estimated fair value as a new cost basis
and the amount of the write-down is included in earnings as an impairment charge. To date, only
temporary impairment adjustments have been recorded.
The recent and precipitous decline in the market value of certain securities backed by
residential mortgage loans has led to a large liquidity crisis affecting the broader U.S. housing
market, the financial services industry and global financial markets. Investors holding many of
these and related securities have experienced substantial decreases in asset valuations and
uncertain secondary market liquidity. Furthermore, credit rating authorities have, in many cases,
been slow to respond to the rapid changes in the underlying value of certain securities and
pervasive market illiquidity, regarding these securities.
As a result, this credit crisis may have a potential impact on the determination of the fair
value of financial instruments or possibly require impairments in the future should the value of
certain investments suffer a decline in value which is determined to be other than temporary.
Consistent with the Companys investment policy, the Company does not use derivative financial
instruments in its investment portfolio. The Company regularly invests excess operating cash in
deposits with major financial institutions, money market funds, notes issued by the U.S.
government, as well as fixed income investments and U.S. bond funds both of which can be readily
purchased and sold using established markets. The Company believes that the market risk arising
from its holdings of these financial instruments is mitigated as many of these securities are
either government backed or of the highest credit rating.
The Companys investment portfolio has not been materially adversely impacted by the recent
disruption in the credit markets. However, if there is continued and expanded disruption in the
credit markets, there can be no assurance that the Companys investment portfolio will not be
adversely affected in the future.
-9-
Cash and available for sale securities consisted of the following as of December 31, 2008
and September 30, 2009:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
As of December 31, 2008 |
|
|
|
|
|
|
|
Unrealized |
|
|
Unrealized |
|
|
Fair |
|
|
|
Cost |
|
|
Gain |
|
|
Loss |
|
|
Value |
|
Cash balances |
|
$ |
3,457 |
|
|
$ |
|
|
|
$ |
|
|
|
$ |
3,457 |
|
Money market fund |
|
|
24,616 |
|
|
|
|
|
|
|
|
|
|
|
24,616 |
|
Commercial paper |
|
|
22,343 |
|
|
|
104 |
|
|
|
|
|
|
|
22,447 |
|
U.S. government agency securities |
|
|
58,341 |
|
|
|
449 |
|
|
|
|
|
|
|
58,790 |
|
Asset-based securities |
|
|
7,251 |
|
|
|
|
|
|
|
(34 |
) |
|
|
7,217 |
|
Corporate debt securities |
|
|
4,583 |
|
|
|
17 |
|
|
|
(3 |
) |
|
|
4,597 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
$ |
120,591 |
|
|
$ |
570 |
|
|
$ |
(37 |
) |
|
$ |
121,124 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Included in cash and cash
equivalents |
|
$ |
28,073 |
|
|
$ |
|
|
|
$ |
|
|
|
$ |
28,073 |
|
Included in marketable securities |
|
|
92,518 |
|
|
|
570 |
|
|
|
(37 |
) |
|
|
93,051 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total cash and available for
sale securities |
|
$ |
120,591 |
|
|
$ |
570 |
|
|
$ |
(37 |
) |
|
$ |
121,124 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
As of September 30, 2009 |
|
|
|
|
|
|
|
Unrealized |
|
|
Unrealized |
|
|
Fair |
|
|
|
Cost |
|
|
Gain |
|
|
Loss |
|
|
Value |
|
Cash balances |
|
$ |
6,516 |
|
|
$ |
|
|
|
$ |
|
|
|
$ |
6,516 |
|
Money market fund |
|
|
13,971 |
|
|
|
|
|
|
|
|
|
|
|
13,971 |
|
U.S. government agency securities |
|
|
68,346 |
|
|
|
89 |
|
|
|
(1 |
) |
|
|
68,434 |
|
Certificate of deposit |
|
|
350 |
|
|
|
|
|
|
|
|
|
|
|
350 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
$ |
89,183 |
|
|
$ |
89 |
|
|
$ |
(1 |
) |
|
$ |
89,271 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Included in cash and cash
equivalents |
|
$ |
20,487 |
|
|
$ |
|
|
|
$ |
|
|
|
$ |
20,487 |
|
Included in marketable securities |
|
|
68,696 |
|
|
|
89 |
|
|
|
(1 |
) |
|
|
68,784 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total cash and available for
sale securities |
|
$ |
89,183 |
|
|
$ |
89 |
|
|
$ |
(1 |
) |
|
$ |
89,271 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
All of the Companys available for sale investments as of December 31, 2008 and
September 30, 2009 are due in one year or less.
Unrealized gains and losses are reported as a component of accumulated other
comprehensive gain/loss in stockholders equity. For the year ended December 31, 2008,
unrealized holding gains included in accumulated other comprehensive income was $0.1 million.
For the nine months ended September 30, 2009, unrealized holding losses included in
accumulated other comprehensive income was $0.4 million.
For the year ended December 31, 2008 and the nine months ended September 30, 2009, there
were no realized gains or losses. The cost of securities sold is based on the specific
identification method.
Unrealized loss positions in the available for sale securities as of December 31, 2008 and
September 30, 2009 reflect temporary impairments that have not been recognized and have been in a
loss position for less than twelve months. The fair value of these available for sale securities
in unrealized loss positions was $8.9 million and $3.6 million as of December 31, 2008 and
September 30, 2009, respectively.
The Company classifies its investments at fair value in one of the following three categories:
Level 1 Quoted prices in active markets for identical assets or liabilities that the
Company has the ability to access at the measurement date.
-10-
Level 2 Inputs other than quoted prices in active markets that are observable for
the asset or liability, either directly or indirectly.
Level 3 Inputs that are unobservable for the asset or liability.
The Companys available for sale investment securities are classified within Level 1 or Level
2 of the fair value hierarchy. These investment securities are valued using quoted market prices,
broker or dealer quotations or other observable inputs. A summary of the fair value of the
Companys available for sale investment securities (allocated by Level) as of September 30, 2009
are identified in the following table (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Level 1 |
|
|
Level 2 |
|
|
Total |
|
Cash/Money market funds |
|
$ |
20,487 |
|
|
$ |
|
|
|
$ |
20,487 |
|
U.S. government agency securities |
|
|
|
|
|
|
68,434 |
|
|
|
68,434 |
|
Certificate of deposit |
|
|
|
|
|
|
350 |
|
|
|
350 |
|
|
|
|
|
|
|
|
|
|
|
|
|
$ |
20,487 |
|
|
$ |
68,784 |
|
|
$ |
89,271 |
|
|
|
|
|
|
|
|
|
|
|
Note 3. Stock-Based Compensation
During the three and nine months ended September 30, 2009, the Company recorded compensation
expense of approximately $2.0 million and $6.0 million, respectively. The stock-based compensation
expense had no impact on the Companys cash flows from operations and financing activities. As of
September 30, 2009, the total unrecognized compensation cost related to non-vested stock options
granted was $12.2 million and is expected to be recognized over a weighted average period of 2.5
years.
The fair value of the options granted is estimated on the date of grant using a
Black-Scholes-Merton option pricing model with the following weighted-average assumptions:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months |
|
|
Nine Months |
|
|
Three Months |
|
|
Nine Months |
|
|
|
Ended |
|
|
Ended |
|
|
Ended |
|
|
Ended |
|
|
|
September 30, |
|
|
September 30, |
|
|
September 30, |
|
|
September 30, |
|
|
|
2008 |
|
|
2008 |
|
|
2009 |
|
|
2009 |
|
Expected stock price volatility |
|
|
78.0 |
% |
|
|
78.2 |
% |
|
|
81.3 |
% |
|
|
80.6 |
% |
Risk free interest rate |
|
|
3.6 |
% |
|
|
3.0 |
% |
|
|
2.8 |
% |
|
|
2.2 |
% |
Expected life of options (years) |
|
|
6.25 |
|
|
|
6.25 |
|
|
|
6.25 |
|
|
|
6.25 |
|
Expected annual dividend per
share |
|
$ |
0.00 |
|
|
$ |
0.00 |
|
|
$ |
0.00 |
|
|
$ |
0.00 |
|
A summary of option activities related to the Companys stock options for the nine months
ended September 30, 2009 is as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted |
|
|
|
|
|
|
|
|
|
|
Weighted |
|
|
Average |
|
|
|
|
|
|
Number |
|
|
Average |
|
|
Remaining |
|
|
Aggregate |
|
|
|
of |
|
|
Exercise |
|
|
Contractual |
|
|
Intrinsic |
|
|
|
Shares |
|
|
Price |
|
|
Life |
|
|
Value |
|
|
|
(in thousands) |
|
|
|
|
|
|
|
|
|
|
(in millions) |
|
Balance at December 31, 2008 |
|
|
3,077.3 |
|
|
$ |
9.19 |
|
|
|
|
|
|
|
|
|
Options granted |
|
|
1,075.1 |
|
|
$ |
10.10 |
|
|
|
|
|
|
|
|
|
Options exercised |
|
|
(15.1 |
) |
|
$ |
4.36 |
|
|
|
|
|
|
|
|
|
Options forfeited |
|
|
(171.8 |
) |
|
$ |
10.26 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at September 30, 2009 |
|
|
3,965.5 |
|
|
$ |
9.40 |
|
|
7.7 years |
|
$ |
4.5 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Vested and unvested expected to
vest, September 30, 2009 |
|
|
3,769.1 |
|
|
$ |
9.35 |
|
|
7.7 years |
|
$ |
4.4 |
|
Exercisable at September 30, 2009 |
|
|
1,948.1 |
|
|
$ |
8.19 |
|
|
6.7 years |
|
$ |
4.1 |
|
-11-
Note 4. Basic and Diluted Net Loss Attributable to Common Stockholders per Common Share
The Company calculates net loss per share as a measurement of the Companys performance while
giving effect to all dilutive potential common shares that were outstanding during the reporting
period. The Company has a net loss for all periods presented; accordingly, the inclusion of common
stock options would be anti-dilutive. Therefore, the weighted average shares used to calculate
both basic and diluted earnings per share are the same.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
|
Nine Months Ended |
|
|
|
September 30, |
|
|
September 30, |
|
(In thousands, except per share amounts) |
|
2008 |
|
|
2009 |
|
|
2008 |
|
|
2009 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Statement of Operations |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss attributable to common stockholders |
|
$ |
(8,180 |
) |
|
$ |
(13,429 |
) |
|
$ |
(25,205 |
) |
|
$ |
(39,523 |
) |
Net loss attributable to common
stockholders per common
share basic and diluted |
|
$ |
(0.36 |
) |
|
$ |
(0.59 |
) |
|
$ |
(1.12 |
) |
|
$ |
(1.75 |
) |
Note 5. Comprehensive Loss
The components of comprehensive loss are as follows (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
|
Nine Months Ended |
|
|
|
September 30, |
|
|
September 30, |
|
|
|
2008 |
|
|
2009 |
|
|
2008 |
|
|
2009 |
|
Net loss |
|
$ |
(8,180 |
) |
|
$ |
(13,429 |
) |
|
$ |
(25,205 |
) |
|
$ |
(39,523 |
) |
Change in
unrealized net gain
on marketable
securities |
|
|
(250 |
) |
|
|
(57 |
) |
|
|
(340 |
) |
|
|
(445 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Comprehensive loss |
|
$ |
(8,430 |
) |
|
$ |
(13,486 |
) |
|
$ |
(25,545 |
) |
|
$ |
(39,968 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Accumulated other comprehensive loss equals the unrealized net gains and losses on marketable
securities which are the only components of other comprehensive loss included in the Companys
financial statements.
Note 6. Capital Structure
Common Stock
As of September 30, 2009, the Company was authorized to issue 50,000,000 shares of common
stock. Dividends on common stock will be paid when, and if declared by the board of
directors. Each holder of common stock is entitled to vote on all matters and is entitled to
one vote for each share held.
Note 7. Development and Commercialization Agreement with Shire
In November 2007, the Company entered into a License and Collaboration Agreement
with Shire. Under the agreement, the Company and Shire were jointly developing the Companys
three lead pharmacological chaperone compounds for lysosomal storage disorders: Amigal,
Plicera and AT2220. The Company granted Shire the rights to commercialize these products
outside the U.S. and retained all rights to its other programs and to develop and
commercialize Amigal, Plicera and AT2220 in the U.S. As discussed below, in October 2009, the
Company and Shire mutually agreed to terminate the collaboration agreement.
The Company received an initial, non-refundable license fee payment of $50 million from
Shire. Joint development costs toward conduct of clinical trials and pursuing global approval
of the three compounds were
being shared 50/50. In addition, the Company was eligible to receive milestone payments
if certain clinical, regulatory and sales-based milestones were met. The Company was also
eligible to receive tiered double-digit royalties on net sales of the products marketed
outside of the U.S.
-12-
As previously noted in Note 1. Description of Business and Significant Accounting
Policies, on October 29, 2009, the Company and Shire agreed to mutually terminate the
collaboration agreement upon concluding that it is in their respective best interests to no
longer collaborate on the development of the Companys three lead pharmacological chaperone
compounds for the treatment of lysosomal storage disorders. As a result of this termination,
Amicus has reacquired all global development and commercialization rights from Shire for these
lead programs and now owns worldwide rights to them. Shire paid the Company $5.2 million
as full and final payment for amounts due to the Company under the
collaboration agreement, and both parties are relieved of all other future obligations
thereunder, financial or otherwise.
The Company had previously determined that its various deliverables due under the
collaboration agreement represent a single unit of accounting for revenue recognition
purposes. The initial, non-refundable upfront license fee payment of $50 million was being
recognized on a straight line basis as Collaboration Revenue over the period of the
performance obligations, which the Company had to be 18 years as contractually defined.
During the three and nine months ended September 30, 2009, the Company recorded $0.7 million
and $2.1 million, respectively, in Collaboration Revenue. As of September 30, 2009, the Company
recorded $2.8 million of current deferred revenue and $42.0 million of long-term deferred revenue
related to the $50 million upfront payment.
During the three and nine months ended September 30, 2008, the Company recorded $0.7 million
and $2.1 million, respectively, in Collaboration Revenue. As of September 30, 2008, the Company
recorded $2.8 million of current deferred revenue and $44.7 million of long-term deferred revenue
related to the $50 million upfront payment.
During the three and nine months ended September 30, 2009, the Company recorded $4.2 million
and $12.8 million, respectively, in Research Revenue. As of September 30, 2009, the Company
recorded $0.5 million of other receivables related to amount due from Shire for reimbursable
research and development costs.
During the three and nine months ended September 30, 2008, the Company recorded $3.0
million and $8.5 million, respectively, in Research Revenue. As of September 30, 2008, the
Company recorded $1.2 million of current portion of deferred revenue related to reimbursed
research and development costs.
Note 8. Short-Term Borrowings and Long-Term Debt
In May 2009, the Company entered into a loan and security agreement with Silicon Valley
Bank that provides for up to $4 million of equipment financing through October 2012.
Borrowings under the loan agreement are collateralized by equipment purchased with the
proceeds of the loan and bear interest at a fixed rate of approximately 9%. The loan
agreement contains customary terms and conditions, including a financial covenant whereby the
Company must maintain a minimum amount of liquidity measured at the end of each month equal to
the greater of (i) $30 million of unrestricted cash, cash equivalents, and marketable
securities, or (ii) six months of trailing cash burn net of outstanding borrowings under the
loan agreement. The Company has at all times been in compliance with this covenant during the
term of the agreement.
At September 30, 2009, the current and long-term amounts due under the loan agreement
were $1.1 million and $2.6 million, respectively. The carrying amount of the Companys
borrowings approximates fair value at September 30, 2009.
-13-
|
|
|
ITEM 2. |
|
MANAGEMENTS DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS |
Overview
We are a clinical-stage biopharmaceutical company focused on the discovery, development
and commercialization of novel small molecule, orally-administered drugs, known as
pharmacological chaperones, for the treatment of a range of human genetic diseases. Certain
human diseases result from mutations in specific genes that, in many cases, lead to the
production of proteins with reduced stability. Proteins with such mutations may not fold into
their correct three-dimensional shape and are generally referred to as misfolded proteins.
Misfolded proteins are often recognized by cells as having defects and, as a result, may be
eliminated prior to reaching their intended location in the cell. The reduced biological
activity of these proteins leads to impaired cellular function and ultimately to disease. Our
novel approach to the treatment of human genetic diseases consists of using pharmacological
chaperones that selectively bind to the target protein increasing the stability of the protein
and helping it fold into the correct three-dimensional shape. This allows proper trafficking
of the protein, thereby increasing protein activity, improving cellular function and
potentially reducing cell stress. We continue to develop our product candidates and explore
new uses for our platform pharmacological chaperone technology.
We have three compounds in clinical development: Amigal (migalastat hydrochloride) for the
treatment of Fabry disease, Plicera (afegostat tartrate) for the treatment of Gaucher disease and
AT2220 (1-deoxynojirimycin HCl) for the treatment of Pompe disease.
Amigal: In the second quarter of 2009, Amicus announced that the Company
reached agreement with the U.S. Food and Drug Administration (FDA) on the key protocol design
elements of its pivotal trial, including the use of the surrogate primary endpoint of the change
in the amount of kidney interstitial capillary GL-3, the substrate that accumulates in the cells
of Fabry patients. In addition, the FDA is in agreement that the Company is eligible to seek
Accelerated Approval for Amigal according to Subpart H regulations. Amicus began submitting the
Phase 3 protocol to investigational sites worldwide in June 2009 and patient enrollment and
dosing are now underway. Furthermore, Amicus previously reported that it completed a series of
discussions with the European Medicines Agency regarding the clinical study required for Amigal
registration in Europe. The Company will provide an update on the timing of the initiation of
this study in 2010.
Plicera: The Company previously reported preliminary results from its Phase 2
randomized, open-label study to assess the safety, tolerability and preliminary efficacy of its
investigational drug, Plicera, in treatment-naive adult patients with type 1 Gaucher disease. Two
dose regimens of Plicera (225 mg three days on/four days off and seven days on/seven days off)
were studied during this six month trial. While all patients enrolled experienced an increase in
the level of the target enzyme (GCase) as measured in white blood cells, clinically meaningful
improvements in key measures of disease were observed in just one of the eighteen patients who
completed the study. The preliminary results suggest that treatment with Plicera was generally
well tolerated, with no serious adverse events reported. Nineteen subjects were enrolled and 18
subjects completed the study. One subject discontinued treatment because of an adverse event
(conjunctivitis-related symptoms). The Company plans to further analyze and evaluate the results
of this Phase 2 study. However, Amicus no longer plans to advance Plicera into Phase 3
development.
AT2220: Late in the third quarter, the Company announced its plans to initiate a Phase 1
study of AT2220 (1-deoxynojirimycin HCl), its investigational drug in development for the
treatment of Pompe Disease. The primary objective of this study is to evaluate the
pharmacokinetics of AT2220 in muscle tissue in healthy adult subjects. The FDA agreed to Amicus
proposal for the Phase 1 study and subsequently converted the clinical hold of AT2220 to a
partial hold to allow the conduct of this study. This open label, single dose Phase 1 study was
initiated in early October and the Company expects to announce results from the trial in the
first half of 2010. Additionally, Amicus continues to be encouraged by the results of
preclinical studies designed to evaluate the use of AT2220 in combination with enzyme replacement
therapy. The Company expects to report additional data from these studies at scientific
conferences in 2010.
-14-
Research: Amicus
continues to invest in research to assess the potential for applying
its chaperone technology platform to the treatment of a broad range of human genetic
diseases. As part of this effort, Amicus continues to conduct preclinical studies in Parkinsons
disease and is investing in new research aimed at evaluating disease targets for other
neurodegenerative and genetic disorders.
Costs associated with the clinical development of Amigal, Plicera and AT2220 and research
conducted on other programs have caused us to generate significant losses to date, which we
expect to continue. These activities are budgeted to expand over time and will require
further resources if we are to be successful. From our inception in February 2002 through
September 30, 2009, we have accumulated a deficit of $203.7 million. As we have not yet
generated commercial sales revenue from any of our product candidates, our operating losses
will continue and are likely to be substantial over the next several years and we may need to
obtain additional funds to further develop our research and development programs and product
candidates. In addition, we will no longer receive cost sharing revenue and no longer be
eligible to receive milestone payments from Shire in connection with our prior collaboration
agreement. For further information, see Note 7. Development and
Commercialization Agreement with Shire.
In October, we announced a work-force reduction of approximately 20 percent, or 26
employees, as a part of a corporate restructuring with reductions occurring across all levels
and organizations within the Company. In addition, we are terminating our relationships with
approximately 17 contractors currently working at the Company. We are taking this step to
reduce costs and to align resources with our key strategic priorities. We estimate that we
will record charges of approximately $0.9 million during the fourth quarter of 2009 for
employment termination costs payable in cash in connection with the workforce reduction.
Collaboration with Shire
On November 7, 2007, we entered into a license and collaboration agreement with Shire.
Under the agreement, Amicus and Shire were jointly developing Amicus three lead
pharmacological chaperone compounds for lysosomal storage disorders: Amigal, Plicera and
AT2220. We granted Shire the rights to commercialize these products outside the United States
(U.S.) and retained all rights to our other programs and to develop and commercialize Amigal,
Plicera and AT2220 in the U.S. In October 2009, the Company and Shire mutually agreed to
terminate the collaboration agreement. For further information, see Note 7.
Development and Commercialization Agreement with Shire.
Financial Operations Overview
Revenue
In connection with our collaboration agreement with Shire, Shire paid us an initial,
non-refundable license fee of $50 million and reimburses us for certain research and
development costs associated with our lead clinical development programs.
For the three and nine months ended September 30, 2009, we recognized approximately $0.7
million and $2.1 million, respectively, of the license fee in Collaboration Revenue and $4.2
million and $12.8 million, respectively, of Research Revenue for reimbursed research and
development costs.
For the three and nine months ended September 30, 2008, we recognized approximately $0.7
million and $2.1 million, respectively, of the license fee in Collaboration Revenue and $3.0
million and $8.5 million, respectively, of Research Revenue for reimbursed research and
development costs.
Research and Development Expenses
We expect our research and development expense to increase as we continue to develop our
product candidates and explore new uses for our pharmacological chaperone technology. Research
and development expense consists of:
|
|
|
internal costs associated with our research and clinical development
activities; |
-15-
|
|
|
payments we make to third party contract research organizations, contract
manufacturers, investigative sites, and consultants; |
|
|
|
technology license costs; |
|
|
|
manufacturing development costs; |
|
|
|
personnel related expenses, including salaries, benefits, travel, and related
costs for the personnel involved in drug discovery and development; |
|
|
|
activities relating to regulatory filings and the advancement of our product
candidates through preclinical studies and clinical trials; and |
|
|
|
facilities and other allocated expenses, which include direct and allocated
expenses for rent, facility maintenance, as well as laboratory and other supplies. |
We have multiple research and development projects ongoing at any one time. We utilize
our internal resources, employees and infrastructure across multiple projects. We record and
maintain information regarding external, out-of-pocket research and development expenses on a
project specific basis.
We expense research and development costs as incurred, including payments made to date
under our license agreements. We believe that significant investment in product development
is a competitive necessity and plan to continue these investments in order to realize the
potential of our product candidates. From our inception in February 2002 through September
30, 2009, we have incurred research and development expense in the aggregate of $165.6
million.
The following table summarizes our principal product development programs, including the
related stages of development for each product candidate in development, and the out-of-pocket,
third party expenses incurred with respect to each product candidate (in thousands).
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Period from |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
February 4, 2002 |
|
|
|
Three Months Ended |
|
|
Nine Months Ended |
|
|
(inception) to |
|
|
|
September 30, |
|
|
September 30, |
|
|
September 30, |
|
Product Candidate |
|
2008 |
|
|
2009 |
|
|
2008 |
|
|
2009 |
|
|
2009 |
|
Third party direct project expenses |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Amigal (Fabry Disease Phase 2) |
|
$ |
906 |
|
|
$ |
3,096 |
|
|
$ |
3,156 |
|
|
$ |
6,854 |
|
|
$ |
32,294 |
|
Plicera (Gaucher Disease Phase 2) |
|
|
547 |
|
|
|
1,098 |
|
|
|
1,572 |
|
|
|
5,693 |
|
|
|
24,597 |
|
AT2220 (Pompe Disease Phase 2) |
|
|
764 |
|
|
|
266 |
|
|
|
1,708 |
|
|
|
1,528 |
|
|
|
12,552 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total third party direct project expenses |
|
|
2,217 |
|
|
|
4,460 |
|
|
|
6,436 |
|
|
|
14,075 |
|
|
|
69,443 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Other project costs (1) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Personnel costs |
|
|
3,454 |
|
|
|
4,830 |
|
|
|
10,423 |
|
|
|
14,759 |
|
|
|
53,725 |
|
Other costs (2) |
|
|
2,529 |
|
|
|
3,319 |
|
|
|
7,130 |
|
|
|
9,120 |
|
|
|
42,428 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total other project costs |
|
|
5,983 |
|
|
|
8,149 |
|
|
|
17,553 |
|
|
|
23,879 |
|
|
|
96,153 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total research and development costs |
|
$ |
8,200 |
|
|
$ |
12,609 |
|
|
$ |
23,989 |
|
|
$ |
37,954 |
|
|
$ |
165,596 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(1) |
|
Other project costs are leveraged across multiple projects. |
|
(2) |
|
Other costs include facility, supply, overhead, and licensing costs that support multiple
clinical and preclinical projects. |
The successful development of our product candidates is highly uncertain. At this time,
we cannot reasonably estimate or know the nature, timing and costs of the efforts that will be
necessary to complete the remainder of the development of our product candidates. As a result, we
are not able to reasonably estimate the period, if any, in which material net cash inflows may
commence from our product candidates, Amigal, Plicera, AT2220 or any of our other preclinical
product candidates. This uncertainty is due to the numerous risks and uncertainties associated
with the conduct, duration and cost of clinical trials, which vary significantly over the life of a
project as a result of evolving events during clinical development, including:
|
|
|
the number of clinical sites included in the trials; |
|
|
|
the length of time required to enroll suitable patients; |
-16-
|
|
|
the number of patients that ultimately participate in the trials; |
|
|
|
the results of our clinical trials; and |
|
|
|
any mandate by the FDA or other regulatory authority to conduct clinical trials
beyond those currently anticipated. |
Our expenditures are subject to additional uncertainties, including the terms and timing of
regulatory approvals, and the expense of filing, prosecuting, defending and enforcing any patent
claims or other intellectual property rights. We may obtain unexpected results from our clinical
trials. We may elect to discontinue, delay or modify clinical trials of some product candidates or
focus on others. A change in the outcome of any of the foregoing variables with respect to the
development of a product candidate could mean a significant change in the costs and timing
associated with the development, regulatory approval and commercialization of that product
candidate. For example, if the FDA or other regulatory authorities were to require us to conduct
clinical trials beyond those which we currently anticipate, or if we experience significant delays
in enrollment in any of our clinical trials, we could be required to expend significant additional
financial resources and time on the completion of clinical development. Drug development may take
several years and millions of dollars in development costs.
General and Administrative Expense
General and administrative expense consists primarily of salaries and other related costs,
including stock-based compensation expense, for persons serving in our executive, finance,
accounting, information technology and human resource functions. Other general and administrative
expense includes facility-related costs not otherwise included in research and development expense,
promotional expenses, costs associated with industry and trade shows, and professional fees for
legal services, including patent-related expense, and accounting services. From our inception in
February 2002 through September 30, 2009, we spent $73.4 million on general and administrative
expense.
Interest Income and Interest Expense
Interest income consists of interest earned on our cash and cash equivalents and marketable
securities. Interest expense consists of interest incurred on our capital lease facility and our
equipment financing agreement.
Critical Accounting Policies and Significant Judgments and Estimates
The discussion and analysis of our financial condition and results of operations are based on
our financial statements, which we have prepared in accordance with U.S. generally accepted
accounting principles. The preparation of these financial statements requires us to make estimates
and assumptions that affect the reported amounts of assets and liabilities and the disclosure of
contingent assets and liabilities at the date of the financial statements, as well as the reported
revenues and expenses during the reporting periods. On an ongoing basis, we evaluate our estimates
and judgments, including those described in greater detail below. We base our estimates on
historical experience and on various other factors that we believe are reasonable under the
circumstances, the results of which form the basis for making judgments about the carrying value of
assets and liabilities that are not readily apparent from other sources. Actual results may differ
from these estimates under different assumptions or conditions.
While there were no significant changes during the quarter ended September 30, 2009 to the
items that we disclosed as our significant accounting policies and estimates described in Note 2 to
the Companys financial statements as contained in the Companys Annual Report on Form 10-K for the
year ended December 31, 2008, we believe that the following accounting policies are the most
critical to aid you in fully understanding and evaluating our financial condition and results of
operations.
Revenue Recognition
The Company recognizes revenue when amounts are realized or realizable and earned.
Revenue is considered realizable and earned when the following criteria are met: (1)
persuasive evidence of an
arrangement exists; (2) delivery has occurred or services have been rendered; (3) the
price is fixed or determinable; and (4) collection of the amounts due are reasonably assured.
-17-
In determining the accounting for collaboration agreements, the Company determines
whether an arrangement involves multiple revenue-generating deliverables that should be
accounted for as a single unit of accounting or divided into separate units of accounting for
revenue recognition purposes. If this division is required, the arrangement consideration
should be allocated among the separate units of accounting. If the arrangement represents a
single unit of accounting, the revenue recognition policy and the performance obligation
period must be determined (if not already contractually defined) for the entire arrangement.
If the arrangement represents separate units of accounting according to the separation
criteria, a revenue recognition policy must be determined for each unit. Revenues for
non-refundable upfront license fee payments will be recognized on a straight line basis as
Collaboration Revenue over the period of the performance obligations.
The revenue associated with reimbursements for research and development costs under
collaboration agreements is included in Research Revenue and the costs associated with these
reimbursable amounts are included in research and development expenses. The Company records these
reimbursements as revenue and not as a reduction of research and development expenses as the
Company has the risks and rewards as the principal in the research and development activities.
Accrued Expenses
When we are required to estimate accrued expenses because we have not yet been invoiced
or otherwise notified of actual cost, we identify services that have been performed on our behalf
and estimate the level of service performed and the associated cost incurred. The majority of our
service providers invoice us monthly in arrears for services performed. We make estimates of our
accrued expenses as of each balance sheet date in our financial statements based on facts and
circumstances known to us. Examples of estimated accrued expenses include:
|
|
|
fees owed to contract research organizations in connection with preclinical and
toxicology studies and clinical trials; |
|
|
|
fees owed to investigative sites in connection with clinical trials; |
|
|
|
fees owed to contract manufacturers in connection with the production of clinical trial
materials; |
|
|
|
fees owed for professional services, and |
|
|
|
unpaid salaries, wages and benefits. |
Stock-Based Compensation
Effective January 1, 2006, we adopted the fair value method of measuring stock-based
compensation, which requires a public entity to measure the cost of employee services received in
exchange for an award of equity instruments based on the grant-date fair value of the award. We
chose the straight-line attribution method for allocating compensation costs and recognized the
fair value of each stock option on a straight-line basis over the requisite service period of the
last separately vesting portion of each award. Expected volatility was calculated based on a
blended weighted average of historical information of our stock and the weighted average of
historical information of similar public entities for which historical information was available.
The average expected life was determined using the mid-point between the vesting date and the end
of the contractual term. The risk-free interest rate is based on U.S. Treasury, zero-coupon issues
with a remaining term equal to the expected life assumed at the date of grant.
We valued the equity instruments, consisting of stock options, issued to non-employees using
the Black-Scholes-Merton valuation model. The measurement of stock-based compensation is subject
to periodic adjustments as the underlying equity instruments vest.
Basic and Diluted Net Loss Attributable to Common Stockholders per Common Share
We calculated net loss per share as a measurement of the Companys performance while
giving effect to all dilutive potential common shares that were outstanding during the reporting
period. We had a net loss for all
periods presented; accordingly, the inclusion of common stock options would be anti-dilutive.
Therefore, the weighted average shares used to calculate both basic and diluted earnings per share
are the same.
-18-
The following table provides a reconciliation of the numerator and denominator used in
computing basic and diluted net loss attributable to common stockholders per common share and pro
forma net loss attributable to common stockholders per common share:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
|
Nine Months Ended |
|
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September 30, |
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September 30, |
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(In thousands, except per share amount) |
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2008 |
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2009 |
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2008 |
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2009 |
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Historical |
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Numerator: |
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Net loss attributable to common
stockholders |
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$ |
(8,180 |
) |
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$ |
(13,429 |
) |
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$ |
(25,205 |
) |
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$ |
(39,523 |
) |
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Denominator: |
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Weighted average common shares
outstanding basic and diluted |
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22,517,431 |
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22,621,513 |
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22,465,981 |
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22,617,808 |
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Dilutive common stock equivalents would include the dilutive effect of common stock options
for common stock equivalents. Potentially dilutive common stock equivalents totaled approximately
3.1 million and 4.0 million for the nine months ended September 30, 2008 and 2009, respectively.
Potentially dilutive common stock equivalents were excluded from the diluted earnings per share
denominator for all periods because of their anti-dilutive effect.
Results of Operations
Three Months Ended September 30, 2009 Compared to Three Months Ended September 30, 2008
Research and Development Expense. Research and development expense was $12.6 million for the
three months ended September 30, 2009 representing an increase of approximately $4.4 million or 54%
from $8.2 million for the three months ended September 30, 2008. The variance was primarily
attributable to higher personnel costs associated with headcount growth, an increase in
manufacturing costs due to the timing of batch production and an increase in contract research
related to clinical trials. We expect research and development expense to continue to increase in
2009 as we move forward with clinical trials relating to our lead clinical development compounds
and expand our discovery research activities.
General and Administrative Expense. General and administrative expense was $5.2 million for
the three months ended September 30, 2009, representing an increase of $0.8 million or 18% from
$4.4 million for the three months ended September 30, 2008. The variance was primarily
attributable higher personnel costs associated with headcount growth.
Interest Income and Interest Expense. Interest income was $0.1 million for the three months
ended September 30, 2009, compared to $1.0 million for the three months ended September 30, 2008.
The decrease of $0.9 million or 90% was due to lower interest rates and decreased cash and cash
equivalents balances. Interest expense was approximately $0.1 million for the three months ended
September 30, 2009 and 2008.
Nine Months Ended September 30, 2009 Compared to Nine Months Ended September 30, 2008
Research and Development Expense. Research and development expense was $38.0 million for the
nine months ended September 30, 2009 representing an increase of approximately $14.0 million or 58%
from $24.0 million for the nine months ended September 30, 2008. The variance was primarily
attributable to higher personnel costs associated with headcount growth, an increase in
manufacturing costs due to the timing of batch production and an increase in contract research
related to clinical trials. We expect research and development expense to continue to increase in
2009 as we move forward with clinical trials relating to our lead clinical development compounds
and expand our discovery research activities.
General and Administrative Expense. General and administrative expense was $15.6 million for
the nine months ended September 30, 2009, representing an increase of $0.9 million or 6% from $14.7
million for the nine months ended September 30, 2008. The variance was primarily attributable
higher personnel costs associated with headcount growth.
-19-
Interest Income and Interest Expense. Interest income was $0.9 million for the nine months
ended September 30, 2009, compared to $4.1 million for the nine months ended September 30, 2008.
The decrease of $3.2 million or 78% was due to lower interest rates and decreased cash and cash
equivalents balances. Interest expense was approximately $0.2 million for the nine months ended
September 30, 2009 and 2008.
Liquidity and Capital Resources
Source of Liquidity
As a result of our significant research and development expenditures and the lack of any
approved products to generate product sales revenue, we have not been profitable and have
generated operating losses since our inception in 2002. We have funded our operations
principally with $148.7 million of proceeds from redeemable convertible preferred stock
offerings, $75.0 million of gross proceeds from our initial public offering in June 2007 and
$50.0 million from the non-refundable license fee from the Shire collaboration agreement in
November 2007. The following table summarizes our significant funding sources as of September
30, 2009:
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Approximate |
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Amount(1) |
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Funding |
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Year |
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No. Shares |
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(in thousands) |
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Series A Redeemable
Convertible Preferred Stock |
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2002 |
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444,443 |
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$ |
2,500 |
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Series B Redeemable
Convertible Preferred Stock |
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2004, 2005, 2006, 2007 |
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4,917,853 |
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31,189 |
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Series C Redeemable
Convertible Preferred Stock |
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2005, 2006 |
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5,820,020 |
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54,999 |
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Series D Redeemable
Convertible Preferred Stock |
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2006, 2007 |
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4,930,405 |
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60,000 |
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Common Stock |
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2007 |
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5,000,000 |
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75,000 |
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Upfront License Fee from Shire |
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2007 |
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50,000 |
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21,112,721 |
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$ |
273,688 |
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(1) |
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Represents gross proceeds |
In addition, in conjunction with the Shire collaboration agreement, we have received
reimbursement of research and development expenditures from the date of the agreement
(November 7, 2007) through September 30, 2009 of $25.9 million. However, we will not receive
any further reimbursement payments from Shire following the mutual termination of our
collaboration agreement with Shire on October 29, 2009.
In May 2009, the Company entered into a loan and security agreement with Silicon Valley
Bank that provides for up to $4 million of equipment financing through October 2012. At
September 30, 2009, the total amount due under the loan agreement was $3.7 million.
As of September 30, 2009, we had cash, cash equivalents and marketable securities of $89.3
million. We invest cash in excess of our immediate requirements with regard to liquidity and
capital preservation in a variety of interest-bearing instruments, including obligations of U.S.
government agencies and money market accounts. Wherever possible, we seek to minimize the
potential effects of concentration and degrees of risk. Although we maintain cash balances with
financial institutions in excess of insured limits, we do not anticipate any losses with respect to
such cash balances.
Net Cash Used in Operating Activities
Net cash used in operations for the nine months ended September 30, 2008 was $22.6
million due to the net loss for the nine months ended September 30, 2008 of $25.2 million, a
reduction in deferred revenue of $1.8 million and the change in other operating assets and
liabilities of $1.4 million.
-20-
Net cash used in operations for the nine months ended September 30, 2009 of $32.8 million was
comprised of the net loss for the nine months ended September 30, 2009 of $39.5 million and a
reduction in deferred revenue of $3.0 million, partially offset by the change in other operating
assets and liabilities of $2.1 million.
Net Cash (Used in)/Provided By Investing Activities
Net cash used in investing activities for the nine months ended September 30, 2008 was $1.1
million. Net cash used in investing activities reflects $125.3 million for the purchase of
marketable securities and $1.7 million for the acquisition of property and equipment, partially
offset by $125.9 million for the sale and redemption of marketable securities.
Net cash provided by investing activities for the nine months ended September 30, 2009 was
$22.1 million. Net cash provided by investing activities reflects $106.4 million for the sale and
redemption of marketable securities, partially offset by $82.6 million for the purchase of
marketable securities and $1.7 million for the acquisition of property and equipment.
Net Cash (Used in)/Provided By Financing Activities
Net cash used in financing activities for the nine months ended September 30, 2008 was
$0.7 million, consisting primarily of $1.1 million of payments of capital lease obligations,
partially offset by $0.4 million of proceeds from exercise of stock options.
Net cash provided by financing activities for the nine months ended September 30, 2009
was $3.1 million and reflected the proceeds from our secured loan agreement of $3.7 million,
partially offset by the payments of our capital lease obligations of $0.7 million.
Funding Requirements
We expect to incur losses from operations for the foreseeable future primarily due to
increasing research and development expenses, including expenses related to the hiring of
personnel and additional clinical trials, and greater general and administrative expenses
resulting from expanding our finance and administrative staff, adding infrastructure, and
incurring additional costs related to being a public company. Our future capital requirements
will depend on a number of factors, including:
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the progress and results of our clinical trials of Amigal, Plicera and AT2220; |
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the scope, progress, results and costs of preclinical development, laboratory
testing and clinical trials for our product candidates; |
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the costs, timing and outcome of regulatory review of our product candidates; |
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the number and development requirements of other product candidates that we pursue; |
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the costs of commercialization activities, including product marketing sales and
distribution; |
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the emergence of competing technologies and other adverse market developments; |
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the costs of preparing, filing and prosecuting patent application and maintaining,
enforcing and defending intellectual property related claims; |
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the extent to which we acquire or invest in businesses, products and technologies; |
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our ability to execute our operational and business plans and realize reductions in
our expenses in line with our restructuring plan; and |
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our ability to establish collaborations and obtain milestone, royalty or other
payments from any such collaborators. |
We do not anticipate that we will generate revenue from commercial sales for at least the next
several years, if at all. In the absence of additional funding, we expect our continuing operating
losses to result in increases in our cash used in operations over the next several quarters and
years. However, we believe that our existing cash and cash equivalents and short-term investments
will be sufficient to enable us to fund our operating expenses and capital expenditure requirements
at least until the second half of 2011.
-21-
Financial Uncertainties Related to Potential Future Payments
Milestone Payments
We have acquired rights to develop and commercialize our product candidates through
licenses granted by various parties. While our license agreements for Amigal and AT2220 do
not contain milestone payment obligations, two of our agreements related to Plicera do require
us to make such payments if certain specified pre-commercialization events occur. Upon the
satisfaction of these milestones and assuming successful development of Plicera, we may be
obligated, under the agreements that we have in place, to make future milestone payments
aggregating up to approximately $7.9 million. However, such potential milestone payments are
subject to many uncertain variables that would cause such payments, if any, to vary in size.
The events that trigger these payments include:
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commencement of Phase 3 clinical trials; |
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submission of a new drug application to the FDA or foreign equivalents; and |
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receipt of marketing approval from the FDA or foreign equivalents. |
Royalties
Under our license agreements, if we owe royalties on net sales for one of our products to
more than one licensor, then we have the right to reduce the royalties owed to one licensor
for royalties paid to another. The amount of royalties to be offset is generally limited in
each license and can vary under each agreement. For Amigal and AT2220, we will owe royalties
only to Mt. Sinai School of Medicine (MSSM). We expect to pay royalties to all three
licensors with respect to Plicera. To date, we have not made any royalty payments on sales of
our products and believe we are several years away from selling any products that would
require us to make any such royalty payments.
On October 31, 2008, the Company amended and restated its license agreement with MSSM.
The amended and restated agreement consolidated previous amendments into a single agreement,
clarified the portion of royalties and milestone payments the Company receives from Shire that
are payable to MSSM, and provided the Company with the sole right to control the prosecution
of patent rights described in the amended and restated license agreement. Under the terms of
the amended and restated license agreement, the Company agreed to pay MSSM $2.6 million in
connection with the $50 million upfront payment that the Company received in November 2007 and
an additional $2.6 million for the sole right to and control over the prosecution of patent
rights.
Whether we will be obligated to make other milestone or royalty payments in the future is
subject to the success of our product development efforts and, accordingly, is inherently
uncertain.
-22-
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ITEM 3. |
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Quantitative and Qualitative Disclosures about Market Risk |
The recent and precipitous decline in the market value of certain securities backed by
residential mortgage loans has led to a large liquidity crisis affecting the broader U.S. housing
market, the financial services industry and global financial markets. Investors holding many of
these and related securities have experienced substantial decreases in asset valuations and
uncertain secondary market liquidity. Furthermore, credit rating authorities have, in many cases,
been slow to respond to the rapid changes in the underlying value of certain securities and
pervasive market illiquidity, regarding these securities.
As a result, this credit crisis may have a potential impact on the determination of the fair
value of financial instruments or possibly require impairments in the future should the value of
certain investments suffer a decline in value which is determined to be other than temporary.
Consistent with our investment policy, we do not use derivative financial instruments in our
investment portfolio. We regularly invest excess operating cash in deposits with major financial
institutions, money market funds, notes issued by the U.S. government, as well as fixed income
investments and U.S. bond funds both of which can be readily purchased and sold using established
markets. We believe that the market risk arising from our holdings of these financial instruments
is minimal. We currently do not believe that any change in the market value of fixed income
investments in our portfolio is material, nor does it warrant a determination that there was any
other than temporary impairment.
We do not have exposure to market risks associated with changes in interest rates, as we have
no variable interest rate debt outstanding. Although we do not believe we have any material
exposure to market risks associated with interest rates, we may experience reinvestment risk as
fixed income securities mature and are reinvested in securities bearing lower interest rates.
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ITEM 4T. |
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CONTROLS AND PROCEDURES |
As of the end of the period covered by this Quarterly Report on Form 10-Q, an evaluation of
the effectiveness of our disclosure controls and procedures (pursuant to Rule 13a-15(e) and
15d-15(e) under the Securities Exchange Act of 1934, as amended (the Exchange Act)) was carried out
under the supervision of our Principal Executive Officer and Principal Financial Officer, with the
participation of our management. Based on that evaluation, the Principal Executive Officer and the
Principal Financial Officer concluded that, as of the end of such period, our disclosure controls
and procedures are effective in recording, processing, summarizing and reporting, on a timely
basis, information required to be disclosed by us in the reports that we file or submit under the
Exchange Act and are effective in ensuring that information required to be disclosed by us in the
reports that we file or submit under the Exchange Act is accumulated and communicated to our
management, including our Principal Executive Officer and Principal Financial Officer, as
appropriate to allow timely decisions regarding required disclosure.
During the fiscal quarter covered by this report, there has been no change in our internal
control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange
Act) that occurred during the fiscal quarter that has materially affected, or is reasonably likely
to materially affect, our internal control over financial reporting.
PART II. OTHER INFORMATION
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ITEM 1. |
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LEGAL PROCEEDINGS |
We are not a party to any material legal proceedings.
The occurrence of any of the following risks could harm our business, financial condition,
results of operations and/or growth prospects. In that case, the trading price of our common stock
could decline, and you may lose all or part of your investment. Additional risks and uncertainties
not presently known to the Company; or risks that the Company currently considers immaterial, may
also impair the Companys operations.
-23-
Risks Related to Our Financial Position and Need for Additional Capital
We have incurred significant operating losses since our inception. We currently do not, and since
inception never have had, any products available for commercial sale. We expect to incur operating
losses for the foreseeable future and may never achieve or maintain profitability.
Since inception, we have incurred significant operating losses. Our net loss attributable to
common stockholders was $39.4 million, $41.5 million and $65.9 million for the years ended 2008,
2007 and 2006 respectively. As of December 31, 2008, we had an accumulated deficit of $164.2
million. To date, we have financed our operations primarily through private placements of our
redeemable convertible preferred stock, proceeds from our initial public offering and from our
prior collaboration agreement with Shire. We have devoted substantially all of our efforts to
research and development, including our preclinical development activities and clinical trials. We
have not completed development of any drugs. We expect to continue to incur significant and
increasing operating losses for at least the next several years and we are unable to predict the
extent of any future losses as we:
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continue our ongoing Phase 3 clinical trial of Amigal (migalastat hydrochloride) for the
treatment of Fabry disease; |
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continue our ongoing Phase 1 clinical trial of AT2220 for the treatment of Pompe disease
and potentially conduct later-stage clinical trials of AT2220; |
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continue our preclinical studies on the use of pharmacological chaperones for the
treatment of diseases of neurodegeneration; |
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continue our preclinical studies on the combination use of pharmacological chaperones
and enzyme replacement therapy in Pompe disease; |
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continue the research and development of additional product candidates; |
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seek regulatory approvals for our product candidates that successfully complete clinical
trials;and |
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establish a sales and marketing infrastructure to commercialize products for which we
may obtain regulatory approval. |
To become and remain profitable, we must succeed in developing and commercializing drugs
with significant market potential. This will require us to be successful in a range of challenging
activities, including the discovery of product candidates, successful completion of preclinical
testing and clinical trials of our product candidates, obtaining regulatory approval for these
product candidates and manufacturing, marketing and selling those products for which we may obtain
regulatory approval. We are only in the preliminary stages of these activities. We may never
succeed in these activities and may never generate revenues that are large enough to achieve
profitability. Even if we do achieve profitability, we may not be able to sustain or increase
profitability on a quarterly or annual basis. Our failure to become or remain profitable could
depress the market price of our common stock and could impair our ability to raise capital, expand
our business, diversify our product offerings or continue our operations.
We commenced a workforce restructuring in October 2009 to focus our efforts on our key clinical,
research and exploratory development programs and to reduce our overall cash burn rate. Even after
giving effect to this restructuring, we may not have sufficient cash to fully develop and
commercialize our un-partnered product candidates, and the restructuring may impact our ability to
execute our business plan.
In October 2009, we commenced a significant workforce restructuring involving the elimination
of approximately 20% of our positions through layoffs from all departments throughout our
organization, including senior management. Our objective with the restructuring is to reduce our
overall cash burn rate and focus on our key clinical programs while maintaining core research and
exploratory development capability. There can be no assurance that we will be able to reduce
spending as planned or that unanticipated costs will not occur. Our
restructuring efforts to focus on key programs may not prove successful due to a variety of
factors, including, without limitation, risks that a smaller workforce may have difficulty
successfully completing research and development efforts. In addition, we may in the future decide
to restructure operations and reduce expenses further by taking such measures as additional
reductions in our workforce and program spending. Any restructuring places a substantial strain on
remaining management and employees and on operational resources; and there is a risk that our
business will be adversely affected by the diversion of management time to the restructuring
efforts. There can be no assurance that following this restructuring, or any future restructuring,
we will have sufficient cash resources to allow us to fund our operations as planned.
-24-
We will need substantial funding and may be unable to raise capital when needed, which would force
us to delay, reduce or eliminate our product development programs or commercialization efforts.
We expect our research and development expenses to increase in connection with our ongoing
activities, particularly as we continue our Phase 3 clinical trial of Amigal, and for any other
later-stage clinical trials of our product candidates. In addition, subject to obtaining regulatory
approval of any of our product candidates, we expect to incur significant commercialization
expenses for product sales and marketing, securing commercial quantities of product from our
manufacturers and product distribution. In addition, we will no longer receive cost sharing revenue
and no longer be eligible to receive milestone payments from Shire in connection with our prior
collaboration agreement. We currently have no commitments or arrangements for any additional
financing to fund the research and development and commercial launch of our product candidates. We
believe that the net proceeds from our initial public offering, together with our existing cash and
cash equivalents and marketable securities, will be sufficient to enable us to fund our operating
expenses and capital expenditure requirements until at least the second half of 2011. Capital may
not be available when needed on terms that are acceptable to us, or at all, especially in light of
the current challenging economic environment. If adequate funds are not available to us on a timely
basis, we may be required to reduce or eliminate research development programs or commercial
efforts.
Our future capital requirements will depend on many factors, including:
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the progress and results of our clinical trials of Amigal and AT2220; |
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the scope, progress, results and costs of preclinical development, laboratory testing
and clinical trials for our other product candidates; |
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the costs, timing and outcome of regulatory review of our product candidates; |
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the number and development requirements of other product candidates that we pursue; |
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the costs of commercialization activities, including product marketing, sales and
distribution; |
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the emergence of competing technologies and other adverse market developments; |
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the costs of preparing, filing and prosecuting patent applications and maintaining,
enforcing and defending intellectual property related claims; |
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the extent to which we acquire or invest in businesses, products and technologies; and |
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our ability to establish collaborations and obtain milestone, royalty or other payments
from any such collaborators. |
Any capital that we obtain may not be on terms favorable to us or our stockholders or may require
us to relinquish valuable rights.
Until such time, if ever, as we generate product revenue to finance our operations, we expect
to finance our cash needs through public or private equity offerings and debt financings, corporate
collaboration and licensing arrangements and grants from patient advocacy groups, foundations and
government agencies. If we are able to raise
capital by issuing equity securities, our stockholders will experience dilution. Debt
financing, if available, may involve agreements that include covenants limiting or restricting our
ability to take specific actions, such as incurring additional debt, making capital expenditures or
declaring dividends and may include rights that are senior to the holders of our common stock. Any
debt financing or additional equity that we raise may contain terms, such as liquidation and other
preferences, which are not favorable to us or our stockholders. If we raise capital through
additional collaboration and licensing arrangements with third parties, it may be necessary to
relinquish valuable rights to our technologies, future revenue streams, research programs or
product candidates or to grant licenses on terms that may not be favorable to us or our
stockholders.
-25-
Our short operating history may make it difficult to evaluate the success of our business to date
and to assess our future viability.
We are a development stage company. We commenced operations in February 2002. Our operations
to date have been limited to organizing and staffing our company, acquiring and developing our
technology and undertaking preclinical studies and limited clinical trials of our most advanced
product candidates. We have not yet generated any commercial sales for any of our product
candidates. We have not yet demonstrated our ability to successfully complete large-scale, clinical
trials, obtain regulatory approvals, manufacture a commercial-scale product or arrange for a third
party to do so on our behalf, or conduct sales and marketing activities necessary for successful
product commercialization. Consequently, any predictions about our future success or viability may
not be as accurate as they could be if we had a longer operating history.
In addition, as a new business, we may encounter unforeseen expenses, difficulties,
complications, delays and other known and unknown factors. If we are successful in obtaining
marketing approval for any of our lead product candidates, we will need to transition from a
company with a research focus to a company capable of supporting commercial activities. We may not
be successful in such a transition.
Risks Related to the Development and Commercialization of Our Product Candidates
We depend heavily on the success of our most advanced product candidate, Amigal. All of our product
candidates are still in either preclinical or clinical development. Clinical trials of our product
candidates may not be successful. If we are unable to commercialize Amigal, or experience
significant delays in doing so, our business will be materially harmed.
We have invested a significant portion of our efforts and financial resources in the
development of our most advanced product candidates, including Amigal. Our ability to generate
product revenue, which we do not expect will occur for at least the next several years, if ever,
will depend heavily on the successful development and commercialization of these product
candidates. The successful commercialization of our product candidates will depend on several
factors, including the following:
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obtaining supplies of Amigal for completion of our clinical trials on a timely basis; |
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successful enrollment of patients in our clinical trials on a timely basis; |
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successful completion of preclinical studies and clinical trials; |
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obtaining regulatory agreement in the structure and design of our Phase 3 clinical
programs; |
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obtaining marketing approvals from the United States Food and Drug Administration (FDA),
and similar regulatory authorities outside the U.S.; |
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establishing commercial-scale manufacturing arrangements with third party manufacturers
whose manufacturing facilities are operated in compliance with current good manufacturing
practice (cGMP) regulations; |
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launching commercial sales of the product, whether alone or in collaboration with
others; |
-26-
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acceptance of the product by patients, the medical community and third party payors; |
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competition from other companies and their therapies; |
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successful protection of our intellectual property rights from competing products in the
U.S. and abroad; and |
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a continued acceptable safety and efficacy profile of our product candidates following
approval. |
If the market opportunities for our product candidates are smaller than we believe they are, then
our revenues may be adversely affected and our business may suffer.
Each of the diseases that our product candidates are being developed to address is rare. Our
projections of both the number of people who have these diseases, as well as the subset of people
with these diseases who have the potential to benefit from treatment with our product candidates,
are based on estimates.
Currently, most reported estimates of the prevalence of these diseases are based on studies of
small subsets of the population of specific geographic areas, which are then extrapolated to
estimate the prevalence of the diseases in the broader world population. In addition, as new
studies are performed the estimated prevalence of these diseases may change. In fact, as a result
of some recent studies, we believe that previously reported studies do not accurately account for
the prevalence of Fabry disease and that the prevalence of Fabry disease could be many times higher
than previously reported. There can be no assurance that the prevalence of Fabry disease or Pompe
disease in the study populations, particularly in these newer studies, accurately reflects the
prevalence of these diseases in the broader world population.
We estimate the number of potential patients in the broader world population who have those
diseases and may respond to treatment with our product candidates by further extrapolating
estimates of the prevalence of specific types of genetic mutations giving rise to these diseases.
For example, we base our estimate of the percentage of Fabry patients who may respond to treatment
with Amigal on the frequency of missense and other similar mutations that cause Fabry disease
reported in the Human Gene Mutation Database. As a result of recent studies that estimate that the
prevalence of Fabry disease could be many times higher than previously reported, we believe that
the number of patients diagnosed with Fabry disease will increase and estimate that the number of
Fabry patients who may benefit from the use of Amigal is significantly higher than some previously
reported estimates of Fabry disease generally. If our estimates of the prevalence of Fabry disease
or Pompe disease or of the number of patients who may benefit from treatment with our product
candidates prove to be incorrect, the market opportunities for our product candidates may be
smaller than we believe they are, our prospects for generating revenue may be adversely affected
and our business may suffer.
Initial results from a clinical trial do not ensure that the trial will be successful and success
in early stage clinical trials does not ensure success in later-stage clinical trials.
We will only obtain regulatory approval to commercialize a product candidate if we can
demonstrate to the satisfaction of the FDA or the applicable non-U.S. regulatory authority, in
well-designed and conducted clinical trials, that the product candidate is safe and effective and
otherwise meets the appropriate standards required for approval for a particular indication.
Clinical trials are lengthy, complex and extremely expensive processes with uncertain results. A
failure of one or more of our clinical trials may occur at any stage of testing. We have limited
experience in conducting and managing the clinical trials necessary to obtain regulatory approvals,
including approval by the FDA.
Success in preclinical testing and early clinical trials does not ensure that later clinical
trials will be successful, and initial results from a clinical trial do not necessarily predict
final results. We cannot be assured that these trials will ultimately be successful. For example,
the Company previously announced disappointing results of a Phase 2 study of Plicera following
successful Phase 1 and Phase 2 studies. In addition, patients may not be compliant with their
dosing regimen or trial protocols or they may withdraw from the study at any time for any reason.
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Even if our early stage clinical trials are successful, we will need to conduct additional
clinical trials with larger numbers of patients receiving the drug for longer periods for all of
our product candidates before we are able to seek approvals to market and sell these product
candidates from the FDA and regulatory authorities outside the U.S. In addition, each of our
product candidates is based on our pharmacological chaperone technology. To date, we are not aware
that any product based on chaperone technology has been approved by the FDA. As a result, while we
have reached agreement with the FDA on the use of a surrogate primary endpoint in our Phase 3 study
for Amigal, we cannot be sure what endpoints the FDA will require us to measure in later-stage
clinical trials of our other product candidates. If the FDA requires different endpoints than the
endpoints we anticipate using, it may be more difficult for us to obtain, or we may be delayed in
obtaining, FDA approval of our product candidates. If we are not successful in commercializing any
of our lead product candidates, or are significantly delayed in doing so, our business will be
materially harmed.
We have limited experience in conducting and managing the preclinical development activities and
clinical trials necessary to obtain regulatory approvals, including approval by the FDA.
We have limited experience in conducting and managing the preclinical development activities
and clinical trials necessary to obtain regulatory approvals, including approval by the FDA. We
have not obtained regulatory approval nor commercialized any of our product candidates. We are
currently conducting a Phase 3 clinical trial for Amigal and a Phase 1 clinical trial for AT2220
but have not yet completed a Phase 3 clinical trial for any of our product candidates.
Additionally, we are conducting preclinical studies on the combination use of pharmacological
chaperones and enzyme replacement therapy in Pompe disease. Our limited experience might prevent us
from successfully designing or implementing a clinical trial. We have limited experience in
conducting and managing the application process necessary to obtain regulatory approvals and we
might not be able to demonstrate that our product candidates meet the appropriate standards for
regulatory approval. If we are not successful in conducting and managing our preclinical
development activities or clinical trials or obtaining regulatory approvals, we might not be able
to commercialize our lead product candidates, or might be significantly delayed in doing so, which
will materially harm our business.
We may find it difficult to enroll patients in our clinical trials.
Each of the diseases that our lead product candidates are intended to treat is relatively rare
and we expect only a subset of the patients with these diseases to be eligible for our clinical
trials. Given that each of our product candidates is in the early stages of required testing, we
may not be able to initiate or continue clinical trials for each or all of our product candidates
if we are unable to locate a sufficient number of eligible patients to participate in the clinical
trials required by the FDA or other non-U.S. regulatory agencies. The requirements of our clinical
testing mandate that a patient cannot be involved in another clinical trial for the same
indication. We are aware that our competitors have ongoing clinical trials for products that are
competitive with our product candidates and patients who would otherwise be eligible for our
clinical trials may be involved in such testing, rendering them unavailable for testing of our
product candidates. Additionally, many patients with Fabry disease and Pompe disease may already be
receiving existing therapies, such as enzyme replacement therapy, which would render them
ineligible for our current clinical trials if they are not willing to stop receiving such
therapies. Further, if we are required to include patients in our clinical trials who have never
received enzyme replacement therapy, we may experience yet further difficulty and delay enrolling
patients in our trials. Our inability to enroll a sufficient number of patients for any of our
current or future clinical trials would result in significant delays or may require us to abandon
one or more clinical trials altogether.
If our preclinical studies do not produce positive results, if our clinical trials are delayed or
if serious side effects are identified during drug development, we may experience delays, incur
additional costs and ultimately be unable to commercialize our product candidates.
Before obtaining regulatory approval for the sale of our product candidates, we must conduct,
at our own expense, extensive preclinical tests to demonstrate the safety of our product candidates
in animals, and clinical trials to demonstrate the safety and efficacy of our product candidates in
humans. Preclinical and clinical testing is expensive, difficult to design and implement and can
take many years to complete. A failure of one or more of our preclinical studies or clinical trials
can occur at any stage of testing. We may experience numerous unforeseen events during, or as a
result of, preclinical testing and the clinical trial process that could delay or prevent our
ability to obtain regulatory approval or commercialize our product candidates, including:
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our preclinical tests or clinical trials may produce negative or inconclusive results,
and we may decide, or regulators may require us, to conduct additional preclinical testing
or clinical trials or we may abandon projects that we expect to be promising; |
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regulators or institutional review boards may not authorize us to commence a clinical
trial or conduct a clinical trial at a prospective trial site; |
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conditions imposed on us by the FDA or any non-U.S. regulatory authority regarding the
scope or design of our clinical trials or may require us to resubmit our clinical trial
protocols to institutional review boards for re-inspection due to changes in the regulatory
environment; |
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the number of patients required for our clinical trials may be larger than we anticipate
or participants may drop out of our clinical trials at a higher rate than we anticipate; |
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our third party contractors or clinical investigators may fail to comply with regulatory
requirements or fail to meet their contractual obligations to us in a timely manner; |
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we might have to suspend or terminate one or more of our clinical trials if we, the
regulators or the institutional review boards determine that the participants are being
exposed to unacceptable health risks; |
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regulators or institutional review boards may require that we hold, suspend or terminate
clinical research for various reasons, including noncompliance with regulatory
requirements; |
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the cost of our clinical trials may be greater than we anticipate; |
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the supply or quality of our product candidates or other materials necessary to conduct
our clinical trials may be insufficient or inadequate or we may not be able to reach
agreements on acceptable terms with prospective clinical research organizations; and |
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the effects of our product candidates may not be the desired effects or may include
undesirable side effects or the product candidates may have other unexpected
characteristics. |
If we are required to conduct additional clinical trials or other testing of our product
candidates beyond those that we currently contemplate, if we are unable to successfully complete
our clinical trials or other testing, if the results of these trials or tests are not positive or
are only modestly positive or if there are safety concerns, we may:
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be delayed in obtaining, or may not be able to obtain, marketing approval for one or
more of our product candidates; |
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obtain approval for indications that are not as broad as intended or entirely different
than those indications for which we sought approval; or |
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have the product removed from the market after obtaining marketing approval. |
Our product development costs will also increase if we experience delays in testing or
approvals. We do not know whether any preclinical tests or clinical trials will be initiated as
planned, will need to be restructured or will be completed on schedule, if at all. Significant
preclinical or clinical trial delays also could shorten the patent protection period during which
we may have the exclusive right to commercialize our product candidates. Such delays could allow
our competitors to bring products to market before we do and impair our ability to commercialize
our products or product candidates.
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The commercial success of any product candidates that we may develop, including Amigal and AT2220,
will depend upon the degree of market acceptance by physicians, patients, third party payors and
others in the medical community.
Any products that we bring to the market, including Amigal and AT2220, if they receive
marketing approval, may not gain market acceptance by physicians, patients, third party payors and
others in the medical community. If these products do not achieve an adequate level of acceptance,
we may not generate significant product revenue and we may not become profitable. The degree of
market acceptance of our product candidates, if approved for commercial sale, will depend on a
number of factors, including:
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the prevalence and severity of any side effects, including any limitations or warnings
contained in a products approved labeling; |
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the efficacy and potential advantages over alternative treatments; |
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the pricing of our product candidates; |
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relative convenience and ease of administration; |
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the willingness of the target patient population to try new therapies and of physicians
to prescribe these therapies; |
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the strength of marketing and distribution support and timing of market introduction of
competitive products; |
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publicity concerning our products or competing products and treatments; and |
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sufficient third party insurance coverage or reimbursement. |
Even if a potential product displays a favorable efficacy and safety profile in preclinical
and clinical trials, market acceptance of the product will not be known until after it is launched.
Our efforts to educate the medical community and third party payors on the benefits of our product
candidates may require significant resources and may never be successful. Such efforts to educate
the marketplace may require more resources than are required by the conventional technologies
marketed by our competitors.
If we are unable to obtain adequate reimbursement from governments or third party payors for any
products that we may develop or if we are unable to obtain acceptable prices for those products,
our prospects for generating revenue and achieving profitability will suffer.
Our prospects for generating revenue and achieving profitability will depend heavily upon the
availability of adequate reimbursement for the use of our approved product candidates from
governmental and other third party payors, both in the U.S. and in other markets. Reimbursement by
a third party payor may depend upon a number of factors, including the third party payors
determination that use of a product is:
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a covered benefit under its health plan; |
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safe, effective and medically necessary; |
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appropriate for the specific patient; |
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neither experimental nor investigational.
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Obtaining reimbursement approval for a product from each government or other third party payor
is a time consuming and costly process that could require us to provide supporting scientific,
clinical and cost effectiveness data for the use of our products to each payor. We may not be able
to provide data sufficient to gain acceptance with respect to reimbursement or we might need to
conduct post-marketing studies in order to demonstrate the cost-
effectiveness of any future products to such payors satisfaction. Such studies might require
us to commit a significant amount of management time and financial and other resources. Even when a
payor determines that a product is eligible for reimbursement, the payor may impose coverage
limitations that preclude payment for some uses that are approved by the FDA or non-U.S. regulatory
authorities. In addition, there is a risk that full reimbursement may not be available for high
priced products. Moreover, eligibility for coverage does not imply that any product will be
reimbursed in all cases or at a rate that allows us to make a profit or even cover our costs.
Interim payments for new products, if applicable, may also not be sufficient to cover our costs and
may not be made permanent.
A primary trend in the U.S. healthcare industry and elsewhere is toward cost containment. We
expect recent changes in the Medicare program and increasing emphasis on managed care to continue
to put pressure on pharmaceutical product pricing. For example, the Medicare Prescription Drug
Improvement and Modernization Act of 2003 provides a new Medicare prescription drug benefit that
began in 2006 and mandates other reforms. While we cannot predict the full outcome of the
implementation of this legislation, it is possible that the new Medicare prescription drug benefit,
which will be managed by private health insurers and other managed care organizations, will result
in additional government reimbursement for prescription drugs, which may make some prescription
drugs more affordable but may further exacerbate industry wide pressure to reduce prescription drug
prices. If one or more of our product candidates reaches commercialization, such changes may have a
significant impact on our ability to set a price we believe is fair for our products and may affect
our ability to generate revenue and achieve or maintain profitability.
Governments outside the U.S. tend to impose strict price controls and reimbursement approval
policies, which may adversely affect our prospects for generating revenue.
In some countries, particularly European Union (EU) countries, the pricing of prescription
pharmaceuticals is subject to governmental control. In these countries, pricing negotiations with
governmental authorities can take considerable time (6 to 12 months or longer) after the receipt of
marketing approval for a product. To obtain reimbursement or pricing approval in some countries, we
may be required to conduct a clinical trial that compares the cost effectiveness of our product
candidate to other available therapies. If reimbursement of our products is unavailable or limited
in scope or amount, or if pricing is set at unsatisfactory levels, our prospects for generating
revenue, if any, could be adversely affected and our business may suffer.
If we are unable to establish sales and marketing capabilities or enter into agreements with third
parties to market and sell our product candidates, we may be unable to generate product revenue.
At present, we have no sales or marketing personnel. In order to commercialize any of our
product candidates, we must either acquire or internally develop sales, marketing and distribution
capabilities, or enter into collaborations with partners to perform these services for us. We may
not be able to establish sales and distribution partnerships on acceptable terms or at all, and if
we do enter into a distribution arrangement, our success will be dependent upon the performance of
our partner.
In the event that we attempt to acquire or develop our own in-house sales, marketing and
distribution capabilities, factors that may inhibit our efforts to commercialize our products
without strategic partners or licensees include:
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our inability to recruit and retain adequate numbers of effective sales and marketing
personnel; |
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the inability of sales personnel to obtain access to or persuade adequate numbers of
physicians to prescribe our products; |
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the lack of complementary products to be offered by our sales personnel, which may put
us at a competitive disadvantage against companies with broader product lines; |
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unforeseen costs associated with creating our own sales and marketing team or with
entering into a partnering agreement with an independent sales and marketing organization;
and |
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efforts by our competitors to commercialize products at or about the time when our
product candidates would be coming to market. |
We may co-promote our product candidates in various markets with pharmaceutical and
biotechnology companies in instances where we believe that a larger sales and marketing presence
will expand the market or accelerate penetration. If we do enter into arrangements with third
parties to perform sales and marketing services, our product revenues will be lower than if we
directly sold and marketed our products and any revenues received under such arrangements will
depend on the skills and efforts of others.
We may not be successful in entering into distribution arrangements and marketing alliances
with third parties. Our failure to enter into these arrangements on favorable terms could delay or
impair our ability to commercialize our product candidates and could increase our costs of
commercialization. Dependence on distribution arrangements and marketing alliances to commercialize
our product candidates will subject us to a number of risks, including:
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we may not be able to control the amount and timing of resources that our distributors
may devote to the commercialization of our product candidates; |
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our distributors may experience financial difficulties; |
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business combinations or significant changes in a distributors business strategy may
also adversely affect a distributors willingness or ability to complete its obligations
under any arrangement; and |
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these arrangements are often terminated or allowed to expire, which could interrupt the
marketing and sales of a product and decrease our revenue. |
If we are unable to establish adequate sales, marketing and distribution capabilities, whether
independently or with third parties, we may not be able to generate product revenue and may not
become profitable.
Product liability lawsuits against us could cause us to incur substantial liabilities and to limit
commercialization of any products that we may develop.
We face an inherent risk of product liability exposure related to the testing of our product
candidates in human clinical trials and will face an even greater risk if we commercially sell any
products that we may develop and which are approved for sale. We may be exposed to product
liability claims and product recalls, including those which may arise from misuse or malfunction
of, or design flaws in, such products, whether or not such problems directly relate to the products
and services we have provided. If we cannot successfully defend ourselves against claims that our
product candidates or products caused injuries, we will incur substantial liabilities. Regardless
of merit or eventual outcome, liability claims may result in:
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decreased demand for any product candidates or products that we may develop; |
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damage to our reputation; |
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regulatory investigations that could require costly recalls or product modifications; |
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withdrawal of clinical trial participants; |
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costs to defend the related litigation; |
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substantial monetary awards to trial participants or patients, including awards that
substantially exceed our product liability insurance, which we would then be required to
pay from other sources, if available, and would damage our ability to obtain liability
insurance at reasonable costs, or at all, in the future; |
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the diversion of managements attention from managing our business; and |
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the inability to commercialize any products that we may develop. |
We have liability insurance policies for our clinical trials in the geographies in which we
are conducting trials. The amount of insurance that we currently hold may not be adequate to cover
all liabilities that we may incur. Insurance coverage is increasingly expensive. We may not be able
to maintain insurance coverage at a reasonable cost and we may not be able to obtain insurance
coverage that will be adequate to satisfy any liability that may arise. On occasion, large
judgments have been awarded in class action lawsuits based on drugs that had unanticipated side
effects. A successful product liability claim or a series of claims brought against us could cause
our stock price to fall and, if judgments exceed our insurance coverage, could decrease our
available cash and adversely affect our business.
We face substantial competition which may result in others discovering, developing or
commercializing products before or more successfully than we do.
The development and commercialization of new drugs is highly competitive and competition is
expected to increase. We face competition with respect to our current product candidates and any
products we may seek to develop or commercialize in the future from major pharmaceutical companies,
specialty pharmaceutical companies and biotechnology companies worldwide. For example, several
large pharmaceutical and biotechnology companies currently market and sell products for the
treatment of Fabry disease. These products include Genzyme Corporations
Fabrazyme® and Shire plcs Replagal®. In addition,
Genzyme Corporation and Actelion, Ltd. market and sell Cerezyme® and
Zavesca®, respectively, for the treatment of Gaucher disease, and Genzyme
Corporation markets and sells Myozyme® for the treatment of Pompe disease. We
are also aware of other enzyme replacement and substrate reduction therapies in development by
third parties.
Potential competitors also include academic institutions, government agencies and other public
and private research organizations that conduct research, seek patent protection and establish
collaborative arrangements for research, development, manufacturing and commercialization. Our
competitors may develop products that are more effective, safer, more convenient or less costly
than any that we are developing or that would render our product candidates obsolete or
noncompetitive. Our competitors may also obtain FDA or other regulatory approval for their products
more rapidly than we may obtain approval for ours. We may also face competition from off-label use
of other approved therapies. There can be no assurance that developments by others that will not
render our product candidates obsolete or noncompetitive either during the research phase or once
the products reach commercialization.
We believe that many competitors, including academic institutions, government agencies, public
and private research organizations, large pharmaceutical companies and smaller more focused
companies, are attempting to develop therapies for many of our target indications.
Many of our competitors have significantly greater financial resources and expertise in
research and development, manufacturing, preclinical testing, conducting clinical trials, obtaining
regulatory approvals, prosecuting intellectual property rights and marketing approved products than
we do. Smaller and other early stage companies may also prove to be significant competitors,
particularly through collaborative arrangements with large and established companies. These third
parties compete with us in recruiting and retaining qualified scientific and management personnel,
establishing clinical trial sites and patient registration for clinical trials, as well as in
acquiring technologies complementary to or necessary for our programs or advantageous to our
business. In addition, if we obtain regulatory approvals for our products, manufacturing efficiency
and marketing capabilities are likely to be significant competitive factors. We currently have no
commercial manufacturing capability, sales force or marketing infrastructure. Further, many of our
competitors have substantial resources and expertise in conducting collaborative arrangements,
sourcing in-licensing arrangements and acquiring new business lines or businesses that are greater
than our own.
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Our business activities involve the use of hazardous materials, which require compliance with
environmental and occupational safety laws regulating the use of such materials. If we violate
these laws, we could be subject to significant fines, liabilities or other adverse consequences.
Our research and development programs involve the controlled use of hazardous materials,
including microbial agents, corrosive, explosive and flammable chemicals and other hazardous
compounds in addition to certain biological hazardous waste. Ultimately, the activities of our
third party product manufacturers when a product candidate reaches commercialization will also
require the use of hazardous materials. Accordingly, we are subject to federal, state and local
laws governing the use, handling and disposal of these materials. Although we believe that our
safety procedures for handling and disposing of these materials comply in all material respects
with the standards prescribed by local, state and federal regulations, we cannot completely
eliminate the risk of accidental contamination or injury from these materials. In addition, our
collaborators may not comply with these laws. In the event of an accident or failure to comply with
environmental laws, we could be held liable for damages that result, and any such liability could
exceed our assets and resources or we could be subject to limitations or stoppages related to our
use of these materials which may lead to an interruption of our business operations or those of our
third party contractors. While we believe that our existing insurance coverage is generally
adequate for our normal handling of these hazardous materials, it may not be sufficient to cover
pollution conditions or other extraordinary or unanticipated events. Furthermore, an accident could
damage or force us to shut down our operations. Changes in environmental laws may impose costly
compliance requirements on us or otherwise subject us to future liabilities and additional laws
relating to the management, handling, generation, manufacture, transportation, storage, use and
disposal of materials used in or generated by the manufacture of our products or related to our
clinical trials. In addition, we cannot predict the effect that these potential requirements may
have on us, our suppliers and contractors or our customers.
Risks Related to Our Dependence on Third Parties
Use of third parties to manufacture our product candidates may increase the risk that we will not
have sufficient quantities of our product candidates or such quantities at an acceptable cost, and
clinical development and commercialization of our product candidates could be delayed, prevented or
impaired.
We do not own or operate manufacturing facilities for clinical or commercial production of our
product candidates. We have limited personnel with experience in drug manufacturing and we lack the
resources and the capabilities to manufacture any of our product candidates on a clinical or
commercial scale. We currently outsource all manufacturing and packaging of our preclinical and
clinical product candidates and products to third parties. The manufacture of pharmaceutical
products requires significant expertise and capital investment, including the development of
advanced manufacturing techniques and process controls. Manufacturers of pharmaceutical products
often encounter difficulties in production, particularly in scaling up initial production. These
problems include difficulties with production costs and yields and quality control, including
stability of the product candidate.
We do not currently have any agreements with third party manufacturers for the long-term
commercial supply of any of our product candidates. We may be unable to enter into agreements for
commercial supply with third party manufacturers, or may be unable to do so on acceptable terms.
Even if we enter into these agreements, the manufacturers of each product candidate will be single
source suppliers to us for a significant period of time.
Reliance on third party manufacturers entails risks, to which we would not be subject if we
manufactured product candidates or products ourselves, including:
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reliance on the third party for regulatory compliance and quality assurance; |
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limitations on supply availability resulting from capacity and scheduling constraints of
the third parties; |
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impact on our reputation in the marketplace if manufacturers of our products, once
commercialized, fail to meet the demands of our customers; |
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the possible breach of the manufacturing agreement by the third party because of factors
beyond our control; and |
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the possible termination or non-renewal of the agreement by the third party, based on
its own business priorities, at a time that is costly or inconvenient for us. |
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The failure of any of our contract manufacturers to maintain high manufacturing standards
could result in injury or death of clinical trial participants or patients using products. Such
failure could also result in product liability claims, product recalls, product seizures or
withdrawals, delays or failures in testing or delivery, cost overruns or other problems that could
seriously harm our business or profitability.
Our contract manufacturers will be required to adhere to FDA regulations setting forth cGMP.
These regulations cover all aspects of the manufacturing, testing, quality control and
recordkeeping relating to our product candidates and any products that we may commercialize. Our
manufacturers may not be able to comply with cGMP regulations or similar regulatory requirements
outside the U.S. Our manufacturers are subject to unannounced inspections by the FDA, state
regulators and similar regulators outside the U.S. Our failure, or the failure of our third party
manufacturers, to comply with applicable regulations could result in sanctions being imposed on us,
including fines, injunctions, civil penalties, failure of regulatory authorities to grant marketing
approval of our product candidates, delays, suspension or withdrawal of approvals, license
revocation, seizures or recalls of product candidates or products, operating restrictions and
criminal prosecutions, any of which could significantly and adversely affect regulatory approval
and supplies of our product candidates.
Our product candidates and any products that we may develop may compete with other product
candidates and products for access to manufacturing facilities. There are a limited number of
manufacturers that operate under cGMP regulations and that are both capable of manufacturing for us
and willing to do so. If the third parties that we engage to manufacture products for our
preclinical tests and clinical trials should cease to continue to do so for any reason, we likely
would experience delays in advancing these trials while we identify and qualify replacement
suppliers and we may be unable to obtain replacement supplies on terms that are favorable to us.
Later relocation to another manufacturer will also require notification, review and other
regulatory approvals from the FDA and other regulators and will subject our production to further
cost and instability in the availability of our product candidates. In addition, if we are not able
to obtain adequate supplies of our product candidates or the drug substances used to manufacture
them, it will be more difficult for us to develop our product candidates and compete effectively.
Our current and anticipated future dependence upon others for the manufacture of our product
candidates may adversely affect our future profit margins and our ability to develop product
candidates and commercialize any products that obtain regulatory approval on a timely and
competitive basis.
Materials necessary to manufacture our product candidates may not be available on commercially
reasonable terms, or at all, which may delay the development and commercialization of our product
candidates.
We rely on the manufacturers of our product candidates to purchase from third party suppliers
the materials necessary to produce the compounds for our preclinical and clinical studies and will
rely on these other manufacturers for commercial distribution if we obtain marketing approval for
any of our product candidates. Suppliers may not sell these materials to our manufacturers at the
time we need them or on commercially reasonable terms and all such prices are susceptible to
fluctuations in price and availability due to transportation costs, government regulations, price
controls and changes in economic climate or other foreseen circumstances. We do not have any
control over the process or timing of the acquisition of these materials by our manufacturers.
Moreover, we currently do not have any agreements for the commercial production of these materials.
If our manufacturers are unable to obtain these materials for our preclinical and clinical studies,
product testing and potential regulatory approval of our product candidates would be delayed,
significantly impacting our ability to develop our product candidates. If our manufacturers or we
are unable to purchase these materials after regulatory approval has been obtained for our product
candidates, the commercial launch of our product candidates would be delayed or there would be a
shortage in supply, which would materially affect our ability to generate revenues from the sale of
our product candidates.
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We rely on third parties to conduct certain preclinical development activities and our clinical
trials and those third parties may not perform satisfactorily, including failing to meet
established deadlines for the completion of such activities and trials.
We do not independently conduct certain preclinical development activities of our product
candidates, such as long-term safety studies in animals, or clinical trials for our product
candidates. We rely on, or work in conjunction
with, third parties, such as contract research organizations, medical institutions and
clinical investigators, to perform this function. Our reliance on these third parties for
preclinical and clinical development activities reduces our control over these activities. We are
responsible for ensuring that each of our preclinical development activities and our clinical
trials is conducted in accordance with the applicable general investigational plan and protocols,
however, we have no direct control over these researchers or contractors (except by contract), as
they are not our employees. Moreover, the FDA requires us to comply with standards, commonly
referred to as Good Clinical Practices for conducting, recording and reporting the results of our
preclinical development activities and our clinical trials to assure that data and reported results
are credible and accurate and that the rights, safety and confidentiality of trial participants are
protected. Our reliance on third parties that we do not control does not relieve us of these
responsibilities and requirements. Furthermore, these third parties may also have relationships
with other entities, some of which may be our competitors. If these third parties do not
successfully carry out their contractual duties, meet expected deadlines or conduct our preclinical
development activities or our clinical trials in accordance with regulatory requirements or our
stated protocols, we will not be able to obtain, or may be delayed in obtaining, regulatory
approvals for our product candidates and will not be able to, or may be delayed in our efforts to,
successfully commercialize our product candidates. Moreover, these third parties may be bought by
other entities or they may go out of business, thereby preventing them from meeting their
contractual obligations.
We also rely on other third parties to store and distribute drug supplies for our preclinical
development activities and our clinical trials. Any performance failure on the part of our existing
or future distributors could delay clinical development or regulatory approval of our product
candidates or commercialization of our products, producing additional losses and depriving us of
potential product revenue.
Extensions, delays, suspensions or terminations of our preclinical development activities and
our clinical trials as a result of the performance of our independent clinical investigators and
contract research organizations will delay, and make more costly, regulatory approval for any
product candidates that we may develop. Any change in a contract research organization during an
ongoing preclinical development activity or clinical trial could seriously delay that trial and
potentially compromise the results of the activity or trial.
We may not be successful in maintaining or establishing collaborations, which could adversely
affect our ability to develop and, particularly in international markets, commercialize products.
For each of our product candidates, we are collaborating with physicians, patient advocacy
groups, foundations and government agencies in order to assist with the development of our
products. We plan to pursue similar activities in future programs and plan to evaluate the merits
of retaining commercialization rights for ourselves or entering into selective collaboration
arrangements with leading pharmaceutical or biotechnology companies. We also may seek to establish
collaborations for the sales, marketing and distribution of our products outside the U.S. If we
elect to seek collaborators in the future but are unable to reach agreements with suitable
collaborators, we may fail to meet our business objectives for the affected product or program. We
face, and will continue to face, significant competition in seeking appropriate collaborators.
Moreover, collaboration arrangements are complex and time consuming to negotiate, document and
implement. We may not be successful in our efforts, if any, to establish and implement
collaborations or other alternative arrangements. The terms of any collaboration or other
arrangements that we establish, if any, may not be favorable to us.
Any collaboration that we enter into may not be successful. The success of our collaboration
arrangements, if any, will depend heavily on the efforts and activities of our collaborators. It is
likely that any collaborators of ours will have significant discretion in determining the efforts
and resources that they will apply to these collaborations. The risks that we may be subject to in
possible future collaborations include the following:
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our collaboration agreements are likely to be for fixed terms and subject to termination
by our collaborators in the event of a material breach or lack of scientific progress by
us; |
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our collaborators are likely to have the first right to maintain or defend our
intellectual property rights and, although we would likely have the right to assume the
maintenance and defense of our intellectual property rights if our collaborators do not,
our ability to do so may be compromised by our collaborators acts or omissions; and |
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our collaborators may utilize our intellectual property rights in such a way as to
invite litigation that could jeopardize or invalidate our intellectual property rights or
expose us to potential liability. |
Collaborations with pharmaceutical companies and other third parties often are terminated or
allowed to expire by the other party. Such terminations or expirations may adversely affect us
financially and could harm our business reputation in the event we elect to pursue collaborations
that ultimately expire or are terminated. For example, in October 2009, we and Shire
Pharmaceuticals Ireland Ltd. (Shire) mutually terminated our collaboration agreement pursuant to
which we were jointly developing our three product candidates for the treatment of lysosomal
storage disorders. As a result, Shire will no longer provide us with the substantial funding it
paid during the last two years. ,
Risks Related to Our Intellectual Property
If we are unable to obtain and maintain protection for the intellectual property relating to our
technology and products, the value of our technology and products will be adversely affected.
Our success will depend in large part on our ability to obtain and maintain protection in the
U.S. and other countries for the intellectual property covering or incorporated into our technology
and products. The patent situation in the field of biotechnology and pharmaceuticals generally is
highly uncertain and involves complex legal, technical, scientific and factual questions. We may
not be able to obtain additional issued patents relating to our technology or products. Even if
issued, patents issued to us or our licensors may be challenged, narrowed, invalidated, held to be
unenforceable or circumvented, which could limit our ability to stop competitors from marketing
similar products or reduce the term of patent protection we may have for our products. Changes in
either patent laws or in interpretations of patent laws in the U.S. and other countries may
diminish the value of our intellectual property or narrow the scope of our patent protection.
The degree of future protection for our proprietary rights is uncertain, and we cannot ensure
that:
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we or our licensors were the first to make the inventions covered by each of our pending
patent applications; |
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we or our licensors were the first to file patent applications for these inventions; |
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others will not independently develop similar or alternative technologies or duplicate
any of our technologies; |
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any patents issued to us or our licensors will provide a basis for commercially viable
products, will provide us with any competitive advantages or will not be challenged by
third parties; |
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we will develop additional proprietary technologies that are patentable; |
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we will file patent applications for new proprietary technologies promptly or at all; |
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our patents will not expire prior to or shortly after commencing commercialization of a
product; or |
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the patents of others will not have a negative effect on our ability to do business. |
In addition, we cannot be assured that any of our pending patent applications will result in
issued patents. In particular, we have filed patent applications in the European Patent Office and
other countries outside the U.S. that have not been issued as patents. These pending applications
include, among others, the patent applications we license pursuant to a license agreement with
Mount Sinai School of Medicine of New York University. If patents are not issued in respect of our
pending patent applications, we may not be able to stop competitors from marketing similar products
in Europe and other countries in which we do not have issued patents.
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The patents and patent applications that we own or have licensed relating to use of Amigal
expire in 2018 in the U.S. and the foreign counterparts, if issued, would expire in 2019. Patents
that we own or have licensed relating to
Plicera expire between 2015 and 2016 in the U.S. and in 2015 outside of the U.S. for
composition of matter, and in 2018 in the U.S. for methods of use. We currently have no issued
patents or pending applications covering methods of using Plicera outside of the U.S. Patents and
patent applications that we own or have licensed relating to the use of AT2220 expire in 2018 in
the U.S. Further, we currently do not have composition of matter or method of use protection for
AT2220 outside of the U.S. Where we lack patent protection outside of the U.S., we intend to seek
orphan medicinal product designation and to rely on statutory data exclusivity provisions in
jurisdictions outside the U.S. where such protections are available, including Europe. If we are
unable to obtain such protection outside the U.S., our competitors may be free to use and sell
Plicera and/or AT2220 outside of the U.S. and there will be no liability for infringement or any
other barrier to competition. The patent rights that we own or have licensed relating to our
product candidates are limited in ways that may affect our ability to exclude third parties from
competing against us if we obtain regulatory approval to market these product candidates. In
particular:
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We do not hold composition of matter patents covering Amigal and AT2220. Composition of
matter patents can provide protection for pharmaceutical products to the extent that the
specifically covered compositions are important. For our product candidates for which we do
not hold composition of matter patents, competitors who obtain the requisite regulatory
approval can offer products with the same composition as our products so long as the
competitors do not infringe any method of use patents that we may hold. |
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For some of our product candidates, the principal patent protection that covers or those
we expect will cover, our product candidate is a method of use patent. This type of patent
only protects the product when used or sold for the specified method. However, this type of
patent does not limit a competitor from making and marketing a product that is identical to
our product that is labeled for an indication that is outside of the patented method, or
for which there is a substantial use in commerce outside the patented method. |
Moreover, physicians may prescribe such a competitive identical product for indications other
than the one for which the product has been approved, or off-label indications, that are covered by
the applicable patents. Although such off-label prescriptions may infringe or induce infringement
of method of use patents, the practice is common and such infringement is difficult to prevent or
prosecute.
Our patents also may not afford us protection against competitors with similar technology.
Because patent applications in the U.S. and many other jurisdictions are typically not published
until 18 months after filing, or in some cases not at all, and because publications of discoveries
in the scientific literature often lag behind the actual discoveries, neither we nor our licensors
can be certain that we or they were the first to make the inventions claimed in our or their issued
patents or pending patent applications, or that we or they were the first to file for protection of
the inventions set forth in these patent applications. If a third party has also filed a U.S.
patent application covering our product candidates or a similar invention, we may have to
participate in an adversarial proceeding, known as an interference, declared by the U.S. Patent and
Trademark Office to determine priority of invention in the U.S. The costs of these proceedings
could be substantial and it is possible that our efforts could be unsuccessful, resulting in a loss
of our U.S. patent position.
If we fail to comply with our obligations in our intellectual property licenses with third parties,
we could lose license rights that are important to our business.
We are a party to a number of license agreements including agreements with the Mount Sinai
School of Medicine of New York University, the University of Maryland, Baltimore County and Novo
Nordisk A/S, pursuant to which we license key intellectual property relating to our lead product
candidates. We expect to enter into additional licenses in the future. Under our existing licenses,
we have the right to enforce the licensed patent rights. Our existing licenses impose, and we
expect that future licenses will impose, various diligence, milestone payment, royalty, insurance
and other obligations on us. If we fail to comply with these obligations, the licensor may have the
right to terminate the license, in which event we might not be able to market any product that is
covered by the licensed patents.
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If we are unable to protect the confidentiality of our proprietary information and know-how, the
value of our technology and products could be adversely affected.
We seek to protect our know-how and confidential information, in part, by confidentiality
agreements with our employees, corporate partners, outside scientific collaborators, sponsored
researchers, consultants and other advisors. We also have confidentiality and invention or patent
assignment agreements with our employees and our consultants. If our employees or consultants
breach these agreements, we may not have adequate remedies for any of these breaches. In addition,
our trade secrets may otherwise become known to or be independently developed by others. Enforcing
a claim that a party illegally obtained and is using our trade secrets is difficult, expensive and
time consuming, and the outcome is unpredictable. In addition, courts outside the U.S. may be less
willing to protect trade secrets. Costly and time consuming litigation could be necessary to seek
to enforce and determine the scope of our proprietary rights, and failure to obtain or maintain
trade secret protection could adversely affect our competitive business position.
If we infringe or are alleged to infringe the intellectual property rights of third parties, it
will adversely affect our business.
Our research, development and commercialization activities, as well as any product candidates
or products resulting from these activities, may infringe or be accused of infringing one or more
claims of an issued patent or may fall within the scope of one or more claims in a published patent
application that may subsequently issue and to which we do not hold a license or other rights.
Third parties may own or control these patents or patent applications in the U.S. and abroad. These
third parties could bring claims against us that would cause us to incur substantial expenses and,
if successful against us, could cause us to pay substantial damages. Further, if a patent
infringement suit were brought against us, we or they could be forced to stop or delay research,
development, manufacturing or sales of the product or product candidate that is the subject of the
suit.
No assurance can be given that patents do not exist, have not been filed, or could not be
filed or issued, which contain claims covering our products, technology or methods. Because of the
number of patents issued and patent applications filed in our field, we believe there is a risk
that third parties may allege they have patent rights encompassing our products, technology or
methods.
We are aware, for example, of U.S. patents, and corresponding international counterparts,
owned by third parties that contain claims related to treating protein misfolding. We have received
written notice from one of these third parties indicating that it believes we may need a license to
certain of these patents in order to avoid infringing such patents. If any of these third party
patents were to be asserted against us we do not believe that our proposed products would be found
to infringe any valid claim of these patents. If we were to challenge the validity of any issued
U.S. patent in court, we would need to overcome a presumption of validity that attaches to every
patent. This burden is high and would require us to present clear and convincing evidence as to the
invalidity of the patents claims. There is no assurance that a court would find in our favor on
infringement or validity.
In order to avoid or settle potential claims with respect to any of the patent rights
described above or any other patent rights of third parties, we may choose or be required to seek a
license from a third party and be required to pay license fees or royalties or both. These licenses
may not be available on acceptable terms, or at all. Even if we or our future collaborators were
able to obtain a license, the rights may be nonexclusive, which could result in our competitors
gaining access to the same intellectual property. Ultimately, we could be prevented from
commercializing a product, or be forced to cease some aspect of our business operations, if, as a
result of actual or threatened patent infringement claims, we are unable to enter into licenses on
acceptable terms. This could harm our business significantly.
Others may sue us for infringing their patent or other intellectual property rights or file
nullity, opposition or interference proceedings against our patents, even if such claims are
without merit, which would similarly harm our business. Furthermore, during the course of
litigation, confidential information may be disclosed in the form of documents or testimony in
connection with discovery requests, depositions or trial testimony. Disclosure of our confidential
information and our involvement in intellectual property litigation could materially adversely
affect our business.
There has been substantial litigation and other proceedings regarding patent and other
intellectual property rights in the pharmaceutical and biotechnology industries. In addition to
infringement claims against us, we may become a party to other patent litigation and other
proceedings, including interference proceedings declared by the
U.S. Patent and Trademark Office and opposition proceedings in the European Patent Office,
regarding intellectual property rights with respect to our products and technology. Even if we
prevail, the cost to us of any patent litigation or other proceeding could be substantial.
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Some of our competitors may be able to sustain the costs of complex patent litigation more
effectively than we can because they have substantially greater resources. In addition, any
uncertainties resulting from any litigation could significantly limit our ability to continue our
operations. Patent litigation and other proceedings may also absorb significant management time.
Many of our employees were previously employed at universities or other biotechnology or
pharmaceutical companies, including our competitors or potential competitors. We try to ensure that
our employees do not use the proprietary information or know-how of others in their work for us.
However, we may be subject to claims that we or these employees have inadvertently or otherwise
used or disclosed intellectual property, trade secrets or other proprietary information of any such
employees former employer. Litigation may be necessary to defend against these claims and, even if
we are successful in defending ourselves, could result in substantial costs to us or be distracting
to our management. If we fail to defend any such claims, in addition to paying monetary damages, we
may jeopardize valuable intellectual property rights, disclose confidential information or lose
personnel.
Risks Related to Regulatory Approval of Our Product Candidates
If we are not able to obtain and maintain required regulatory approvals, we will not be able to
commercialize our product candidates, and our ability to generate revenue will be materially
impaired.
Our product candidates, including Amigal, and the activities associated with their development
and commercialization, including their testing, manufacture, safety, efficacy, recordkeeping,
labeling, storage, approval, advertising, promotion, sale and distribution, are subject to
comprehensive regulation by the FDA and other regulatory agencies in the U.S. and by comparable
authorities in other countries. Failure to obtain regulatory approval for a product candidate will
prevent us from commercializing the product candidate in the jurisdiction of the regulatory
authority. We have not obtained regulatory approval to market any of our product candidates in any
jurisdiction. We have only limited experience in filing and prosecuting the applications necessary
to obtain regulatory approvals and expect to rely on third party contract research organizations to
assist us in this process.
Securing FDA approval requires the submission of extensive preclinical and clinical data and
supporting information to the FDA for each therapeutic indication to establish the product
candidates safety and efficacy. Securing FDA approval also requires the submission of information
about the product manufacturing process to, and inspection of manufacturing facilities by, the FDA.
Our future products may not be effective, may be only moderately effective or may prove to have
undesirable or unintended side effects, toxicities or other characteristics that may preclude our
obtaining regulatory approval or prevent or limit commercial use.
Our product candidates may fail to obtain regulatory approval for many reasons, including:
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our failure to demonstrate to the satisfaction of the FDA or comparable regulatory
authorities that a product candidate is safe and effective for a particular indication; |
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the results of clinical trials may not meet the level of statistical significance
required by the FDA or comparable regulatory authorities for approval; |
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our inability to demonstrate that a product candidates benefits outweigh its risks; |
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our inability to demonstrate that the product candidate is at least as effective as
existing therapies; |
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the FDAs or comparable regulatory authorities disagreement with the manner in which we
interpret the data from preclinical studies or clinical trials; |
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the FDAs or comparable regulatory authorities failure to approve the manufacturing
processes, quality procedures or manufacturing facilities of third party manufacturers with
which we contract for clinical or commercial supplies; and |
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a change in the approval policies or regulations of the FDA or comparable regulatory
authorities or a change in the laws governing the approval process. |
The process of obtaining regulatory approvals is expensive, often takes many years, if
approval is obtained at all, and can vary substantially based upon a variety of factors, including
the type, complexity and novelty of the product candidates involved. Changes in regulatory approval
policies during the development period, changes in or the enactment of additional statutes or
regulations, or changes in regulatory review for each submitted product application may cause
delays in the approval or rejection of an application. The FDA and non-U.S. regulatory authorities
have substantial discretion in the approval process and may refuse to accept any application or may
decide that our data is insufficient for approval and require additional preclinical, clinical or
other studies. In addition, varying interpretations of the data obtained from preclinical and
clinical testing could delay, limit or prevent regulatory approval of a product candidate. Any
regulatory approval we ultimately obtain may be limited or subject to restrictions or post approval
commitments that render the approved product not commercially viable. Any FDA or other regulatory
approval of our product candidates, once obtained, may be withdrawn, including for failure to
comply with regulatory requirements or if clinical or manufacturing problems follow initial
marketing.
Our product candidates may cause undesirable side effects or have other properties that could delay
or prevent their regulatory approval or commercialization.
Undesirable side effects caused by our product candidates could interrupt, delay or halt
clinical trials and could result in the denial of regulatory approval by the FDA or other
regulatory authorities for any or all targeted indications, and in turn prevent us from
commercializing our product candidates and generating revenues from their sale. For example, in a
clinical trial of AT2220 for Pompe disease, two patients experienced self-reported adverse events
and subsequently withdrew from the trial. The events were categorized by the site investigator as
serious and probably related to treatment of AT2220. Further, in a clinical trial of Amigal for
Fabry disease, one patient with a history of hypertension experienced increased blood pressure
during the course of the trial which was reported by the investigator as possibly related to the
drug; and Amigal has been shown to cause reversible infertility effects in mice.
In addition, if any of our product candidates receive marketing approval and we or others
later identify undesirable side effects caused by the product:
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regulatory authorities may require the addition of restrictive labeling statements; |
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regulatory authorities may withdraw their approval of the product; and |
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we may be required to change the way the product is administered or conduct additional
clinical trials. |
Any of these events could prevent us from achieving or maintaining market acceptance of the
affected product or could substantially increase the costs and expenses of commercializing the
product candidate, which in turn could delay or prevent us from generating significant revenues
from its sale or adversely affect our reputation.
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We may not be able to obtain orphan drug exclusivity for our product candidates. If our competitors
are able to obtain orphan drug exclusivity for their products that are the same drug as our product
candidates, we may not be able to have competing products approved by the applicable regulatory
authority for a significant period of time.
Regulatory authorities in some jurisdictions, including the U.S. and Europe, may designate
drugs for relatively small patient populations as orphan drugs. We obtained orphan drug
designations from the FDA for Amigal for the treatment of Fabry disease on February 25, 2004, for
the active ingredient in Plicera for the treatment of Gaucher
disease on January 10, 2006 and for AT2220 for the treatment of Pompe disease on June 18,
2007. We also obtained orphan medicinal product designation in the EU for Amigal on May 22, 2006
and for Plicera on October 23, 2007. We anticipate filing for orphan drug designation in the EU for
AT2220 for the treatment of Pompe disease. Generally, if a product with an orphan drug designation
subsequently receives the first marketing approval for the indication for which it has such
designation, the product is entitled to a period of marketing exclusivity, which precludes the
applicable regulatory authority from approving another marketing application for the same drug for
that time period. The applicable period is 7 years in the U.S. and 10 years in Europe. For a drug
composed of small molecules, the FDA defines same drug as a drug that contains the same active
molecule and is intended for the same use. Obtaining orphan drug exclusivity for Amigal and Plicera
may be important to each of the product candidates success. Even if we obtain orphan drug
exclusivity for Amigal or Plicera for these indications, we may not be able to maintain it. For
example, if a competitive product that is the same drug as our product candidate is shown to be
clinically superior to our product candidate, any orphan drug exclusivity we have obtained will not
block the approval of such competitive product and we may effectively lose what had previously been
orphan drug exclusivity.
Any product for which we obtain marketing approval could be subject to restrictions or withdrawal
from the market and we may be subject to penalties if we fail to comply with regulatory
requirements or if we experience unanticipated problems with our products, when and if any of them
are approved.
Any product for which we obtain marketing approval, along with the manufacturing processes,
post approval clinical data, labeling, advertising and promotional activities for such product,
will be subject to continual requirements of and review by the FDA and comparable regulatory
authorities. These requirements include submissions of safety and other post marketing information
and reports, registration requirements, cGMP requirements relating to quality control, quality
assurance and corresponding maintenance of records and documents, requirements regarding the
distribution of samples to physicians and recordkeeping. Even if we obtain regulatory approval of a
product, the approval may be subject to limitations on the indicated uses for which the product may
be marketed or to the conditions of approval, or contain requirements for costly post marketing
testing and surveillance to monitor the safety or efficacy of the product. We also may be subject
to state laws and registration requirements covering the distribution of our products. Later
discovery of previously unknown problems with our products, manufacturers or manufacturing
processes, or failure to comply with regulatory requirements, may result in actions such as:
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restrictions on such products, manufacturers or manufacturing processes; |
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withdrawal of the products from the market; |
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refusal to approve pending applications or supplements to approved applications that we
submit; |
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voluntary or mandatory recall; |
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suspension or withdrawal of regulatory approvals or refusal to approve pending
applications or supplements to approved applications that we submit; |
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refusal to permit the import or export of our products; |
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product seizure or detentions; |
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injunctions or the imposition of civil or criminal penalties; and |
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adverse publicity.
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If we, or our suppliers, third party contractors, clinical investigators or collaborators are
slow to adapt, or are unable to adapt, to changes in existing regulatory requirements or adoption
of new regulatory requirements or policies, we or our collaborators may lose marketing approval for
our products when and if any of them are approved, resulting in decreased revenue from milestones,
product sales or royalties.
Failure to obtain regulatory approval in international jurisdictions would prevent us from
marketing our products abroad.
We intend to have our products marketed outside the U.S. In order to market our products in
the EU and many other jurisdictions, we must obtain separate regulatory approvals and comply with
numerous and varying regulatory requirements. The approval procedures vary among countries and can
involve additional testing and clinical trials. The time required to obtain approval may differ
from that required to obtain FDA approval. The regulatory approval process outside the U.S. may
include all of the risks associated with obtaining FDA approval. In addition, in many countries
outside the U.S., it is required that the product be approved for reimbursement by
government-backed healthcare regulators or insurance providers before the product can be approved
for sale in that country. We may not obtain approvals from regulatory authorities outside the U.S.
on a timely basis, if at all. Approval by the FDA does not ensure approval by regulatory
authorities in other countries or jurisdictions, and approval by one regulatory authority outside
the U.S. does not ensure approval by regulatory authorities in other countries or jurisdictions or
by the FDA. We may not be able to file for regulatory approvals and may not receive necessary
approvals to commercialize our products in any market.
Risks Related to Employee Matters and Managing Our Restructuring
Our future success depends on our ability to retain our Chief Executive Officer and other key
executives and to attract, retain and motivate qualified personnel.
We are highly dependent on John F. Crowley, our President and Chief Executive Officer, Matthew
R. Patterson, our Chief Operating Officer, David J. Lockhart, Ph.D., our Chief Scientific Officer
and Pol F. Boudes, M.D., our Chief Medical Officer. These executives each have significant
pharmaceutical industry experience, including Mr. Crowley, with whom we have entered into an
employment agreement that runs for successive one year terms until either we or Mr. Crowley elect
to terminate the agreement. We may terminate Mr. Crowleys employment without cause at any time, or
we may decide not to extend Mr. Crowleys agreement at the end of any term, or he may terminate his
employment for good reason at any time, in each case subject to certain severance payments and
benefits. Mr. Crowley is a commissioned officer in the U.S. Navy (Reserve). The U.S. recently
called Mr. Crowley to service, which he fulfilled, from September 11, 2006 to March 5, 2007, and he
may be called to active duty service again at any time. The loss of Mr. Crowley for protracted
military duty could materially adversely affect our business. We are also parties to employment
agreements with each of Mr. Patterson, Dr. Lockhart and Dr. Boudes. These employment agreements
each provide for an initial term of two years, and will continue thereafter for successive two-year
periods until we provide the executive with written notice of the end of the agreement in
accordance with its terms. We may terminate any of these executives without cause at any time, or
one of these executives may quit for good reason within six months of the occurrence of certain
corporate changes, in each case subject to certain severance payments and benefits. The loss of the
services of any of these executives might impede the achievement of our research, development and
commercialization objectives and materially adversely affect our business. We do not maintain key
person insurance on Mr. Crowley or on any of our other executive officers.
We have become even more dependent on existing personnel since the significant workforce
restructuring that we commenced in October 2009, involving the elimination of approximately 20% of
our positions through layoffs from all departments throughout our organization, including senior
management. While the restructuring was designed to focus the Company on its key clinical programs
while maintaining core research and exploratory development capability, the restructuring may
adversely affect the pace and breadth of our research and development efforts.
Recruiting and retaining qualified scientific personnel, clinical personnel and sales and
marketing personnel will also be critical to our success. Our industry has experienced a high rate
of turnover in recent years. We may not be able to attract and retain these personnel on acceptable
terms given the competition among numerous pharmaceutical and biotechnology companies for similar
personnel, particularly in New Jersey and surrounding areas. Although we believe we offer
competitive salaries and benefits, we may have to increase spending in order to retain personnel.
Also, when recruiting new personnel, the occurrence of our October 2009 workforce restructuring may
make it more difficult to attract new personnel. If we fail to retain our remaining qualified
personnel or replace them when they leave, we may be unable to continue our development and
commercialization activities.
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We also experience competition for the hiring of scientific and clinical personnel from
universities and research institutions. In addition, we rely on consultants and advisors, including
scientific and clinical advisors, to assist us in formulating our research and development and
commercialization strategy. Our consultants and advisors may be employed by employers other than us
and may have commitments under consulting or advisory contracts with other entities that may limit
their availability to us.
Risks Related to Our Common Stock
Our executive officers, directors and principal stockholders maintain the ability to control all
matters submitted to our stockholders for approval.
Our executive officers, directors and principal stockholders beneficially own shares
representing approximately 67% of our common stock. As a result, if these stockholders were to
choose to act together, they would be able to control all matters submitted to our stockholders for
approval, as well as our management and affairs. For example, these persons, if they choose to act
together, will control the election of directors and approval of any merger, consolidation, sale of
all or substantially all of our assets or other business combination or reorganization. This
concentration of voting power could delay or prevent an acquisition of us on terms that other
stockholders may desire. The interests of this group of stockholders may not always coincide with
the interests of other stockholders, and they may act, whether by meeting or written consent of
stockholders, in a manner that advances their best interests and not necessarily those of other
stockholders, including obtaining a premium value for their common stock, and might affect the
prevailing market price for our common stock.
Provisions in our corporate charter documents and under Delaware law could make an acquisition of
us, which may be beneficial to our stockholders, more difficult and may prevent attempts by our
stockholders to replace or remove our current management.
Provisions in our corporate charter and our bylaws may discourage, delay or prevent a merger,
acquisition or other change in control of us that stockholders may consider favorable, including
transactions in which our stockholders might otherwise receive a premium for their shares. These
provisions could also limit the price that investors might be willing to pay in the future for
shares of our common stock, thereby depressing the market price of our common stock. In addition,
these provisions may frustrate or prevent any attempts by our stockholders to replace or remove our
current management by making it more difficult for stockholders to replace members of our board of
directors. Because our board of directors is responsible for appointing the members of our
management team, these provisions could in turn affect any attempt by our stockholders to replace
current members of our management team. Among others, these provisions:
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establish a classified board of directors, and, as a result, not all directors are
elected at one time; |
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allow the authorized number of our directors to be changed only by resolution of our
board of directors; |
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limit the manner in which stockholders can remove directors from our board of directors; |
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establish advance notice requirements for stockholder proposals that can be acted on at
stockholder meetings and nominations to our board of directors; |
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require that stockholder actions must be effected at a duly called stockholder meeting
and prohibit actions by our stockholders by written consent; |
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limit who may call stockholder meetings; |
-44-
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authorize our board of directors to issue preferred stock, without stockholder approval,
which could be used to institute a poison pill that would work to dilute the stock
ownership of a potential hostile acquirer, effectively preventing acquisitions that have
not been approved by our board of directors; and |
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require the approval of the holders of at least 67% of the votes that all our
stockholders would be entitled to cast to amend or repeal certain provisions of our charter
or bylaws. |
Moreover, because we are incorporated in Delaware, we are governed by the provisions of
Section 203 of the Delaware General Corporation Law, which prohibits a person who owns in excess of
15% of our outstanding voting stock from merging or combining with us for a period of three years
after the date of the transaction in which the person acquired in excess of 15% of our outstanding
voting stock, unless the merger or combination is approved in a prescribed manner.
An active trading market for our common stock may not develop.
We completed our initial public offering of equity securities in June 2007, and prior to this
offering, there was no public market for our common stock. Although we have been listed on The
NASDAQ Global Market, an active trading market for our common stock may never develop or be
sustained. If an active market for our common stock does not develop or is not sustained, it may be
difficult for our stockholders to sell shares since our initial public offering without depressing
the market price for our common stock.
If the price of our common stock is volatile, purchasers of our common stock could incur
substantial losses.
The price of our common stock is volatile. The stock market in general and the market for
biotechnology companies in particular have experienced extreme volatility that has often been
unrelated to the operating performance of particular companies. The market price for our common
stock may be influenced by many factors, including:
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results of clinical trials of our product candidates or those of our competitors; |
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our entry into or the loss of a significant collaboration; |
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regulatory or legal developments in the U.S. and other countries, including changes in
the health care payment systems; |
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variations in our financial results or those of companies that are perceived to be
similar to us; |
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changes in the structure of healthcare payment systems; |
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market conditions in the pharmaceutical and biotechnology sectors and issuance of new or
changed securities analysts reports or recommendations; |
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general economic, industry and market conditions; |
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results of clinical trials conducted by others on drugs that would compete with our
product candidates; |
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developments or disputes concerning patents or other proprietary rights; |
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public concern over our product candidates or any products approved in the future; |
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future sales or anticipated sales of our common stock by us or our stockholders; and |
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the other factors described in this Risk Factors section. |
-45-
For these reasons and others potential purchasers of our common stock should consider an
investment in our common stock as risky and invest only if they can withstand a significant loss
and wide fluctuations in the marked value of their investment.
If securities or industry analysts do not publish research or reports or publish unfavorable
research about our business, the price of our common stock and trading volume could decline.
The trading market for our common stock will depend in part on the research and reports that
securities or industry analysts publish about us or our business. If securities or industry
analysts do not continue coverage of us the trading price for our common stock would be negatively
affected. In the event we obtain securities or industry analyst coverage, if one or more of the
analysts who covers us downgrades our common stock, the price of our common stock would likely
decline. If one or more of these analysts ceases to cover us or fails to publish regular reports on
us, interest in the purchase of our common stock could decrease, which could cause the price of our
common stock or trading volume to decline.
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ITEM 2. |
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UNREGISTERED SALES OF EQUITY SECURITIES AND USE OF PROCEEDS |
Recent Sales of Unregistered Securities
None.
Use of Proceeds
Our initial public offering of common stock was effected through a Registration Statement on
Form S-1 (File No. 333-141700) that was declared effective by the Securities and Exchange
Commission on May 30, 2007, which registered an aggregate of 5,750,000 shares of our common stock.
On June 5, 2007, at the closing of the offering, 5,000,000 shares of common stock were sold on our
behalf at an initial public offering price of $15.00 per share, for aggregate offering proceeds of
$75.0 million. The initial public offering was underwritten and managed by Morgan Stanley, Merrill
Lynch & Co., JPMorgan, Lazard Capital Markets and Pacific Growth Equities, LLC. Following the sale
of the 5,000,000 shares, the public offering terminated.
We paid underwriting discounts totaling approximately $5.3 million and incurred additional
costs of approximately $1.6 million in connection with the offering, for total expenses of
approximately $6.9 million. After deducting underwriting discounts and offering expenses, the net
offering proceeds to us were approximately $68.1 million. No offering expenses were paid directly
or indirectly to any of our directors or officers (or their associates) or persons owning ten
percent or more of any class of our equity securities or to any other affiliates.
As of October 30, 2009, we had invested the $68.1 million in net proceeds from the offering in
money market funds and in investment-grade, interest bearing instruments, pending their use.
Through November 1, 2009, we have not used the net proceeds from the offering. We intend to use
the proceeds for clinical development of our drug candidates, for research and development
activities relating to additional preclinical programs and to fund working capital and other
general corporate purposes, which may include the acquisition or licensing of complementary
technologies, products or businesses.
-46-
Issuer Purchases of Equity Securities
The following table sets forth purchases of our common stock for the three months ended
September 30, 2009:
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(c) Total number of |
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(b) |
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shares purchased as |
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(d) Maximum number of shares |
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(a) Total number |
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Average |
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part of publicly |
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that may yet be |
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of shares |
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Price Paid |
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announced plans or |
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purchased under the plans or |
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Period |
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purchased |
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per Share |
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programs |
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programs |
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July 1,
2009 July 31, 2009 |
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220 |
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$ |
11.45 |
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3,315 |
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August 1, 2009 August 31, 2009 |
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220 |
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$ |
11.39 |
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3,095 |
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September 1, 2009 September
30, 2009 |
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220 |
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$ |
9.83 |
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2,875 |
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Total |
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660 |
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Pursuant to a restricted stock award dated October 2, 2006 between Amicus
Therapeutics and James E. Dentzer, our former Chief Financial Officer, Mr. Dentzer was granted
40,000 restricted shares, 25% of which vested on October 2, 2007. The remaining shares vest
in a series of thirty-six successive equal monthly installments commencing on November 1, 2007
and ending on November 1, 2010. In order to comply with the minimum statutory federal tax
withholding rate of 25% plus 1.45% for Medicare, Mr. Dentzer surrenders to us a portion of his
vested shares on each vesting date, representing 26.45% of the total value of the shares then
vested.
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ITEM 3. |
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DEFAULTS UPON SENIOR SECURITIES |
None.
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ITEM 4. |
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SUBMISSION OF MATTERS TO A VOTE OF SECURITY HOLDERS |
None.
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ITEM 5. |
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OTHER INFORMATION |
None.
-47-
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Exhibit |
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Number |
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Description |
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3.1 (1)
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Restated Certificate of Incorporation |
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3.2 (2)
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Amended and Restated By-laws |
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31.1*
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Certification of Principal Executive Officer pursuant to
Rules 13a-14 and 15d-14 promulgated pursuant to the
Securities Exchange Act of 1934, as amended |
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31.2*
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Certification of Principal Financial Officer pursuant to
Rules 13a-14 and 15d-14 promulgated pursuant to the
Securities Exchange Act of 1934, as amended |
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32.1*
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Certification of Principal Executive Officer and Principal
Financial Officer pursuant to Section 906 of the
Sarbanes-Oxley Act of 2002 |
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(1)
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Incorporated by reference to Exhibit 3.2 to our Registration Statement on Form S-1 |
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(2)
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Incorporated by reference to Exhibit 3.4 to our Registration Statement on Form S-1 |
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* |
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These certifications are being furnished solely to accompany this quarterly
report pursuant to 18 U.S.C. Section 1350, and are not being filed for purposes
of Section 18 of the Securities Exchange Act of 1934 and are not to be
incorporated by reference into any filing of Amicus Therapeutics, Inc., whether
made before or after the date hereof, regardless of any general incorporation
language in such filing. |
-48-
SIGNATURES
Pursuant to the requirements of the Securities and Exchange Act of 1934, the registrant has
duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
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AMICUS THERAPEUTICS, INC.
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Date: November 4, 2009 |
By: |
/s/ JOHN F. CROWLEY
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John F. Crowley |
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President and Chief Executive Officer
(Principal Executive Officer) |
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Date: November 4, 2009 |
By: |
/s/ JOHN M. MCADAM
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John M. McAdam |
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Sr. Director, Finance & Accounting and
Corporate Controller
(Principal Financial Officer) |
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-49-
INDEX TO EXHIBITS
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Exhibit |
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|
Number |
|
Description |
|
|
|
3.1 (1)
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Restated Certificate of Incorporation |
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3.2 (2)
|
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Amended and Restated By-laws |
|
|
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31.1*
|
|
Certification of Principal Executive Officer pursuant to Rules
13a-14 and 15d-14 promulgated pursuant to the Securities
Exchange Act of 1934, as amended |
|
|
|
31.2*
|
|
Certification of Principal Financial Officer pursuant to Rules
13a-14 and 15d-14 promulgated pursuant to the Securities
Exchange Act of 1934, as amended |
|
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|
32.1*
|
|
Certification of Principal Executive Officer and Principal
Financial Officer pursuant to Section 906 of the
Sarbanes-Oxley Act of 2002 |
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(1)
|
|
Incorporated by reference to Exhibit 3.2 to our Registration Statement on Form S-1 |
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(2)
|
|
Incorporated by reference to Exhibit 3.4 to our Registration Statement on Form S-1 |
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* |
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These certifications are being furnished solely to accompany this quarterly
report pursuant to 18 U.S.C. Section 1350, and are not being filed for purposes
of Section 18 of the Securities Exchange Act of 1934 and are not to be
incorporated by reference into any filing of Amicus Therapeutics, Inc., whether
made before or after the date hereof, regardless of any general incorporation
language in such filing. |
-50-