e10vq
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 March 31, 2007
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 000-30171
SANGAMO BIOSCIENCES, INC.
(exact name of small business issuer as specified in its charter)
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Delaware
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68-0359556 |
(State or other jurisdiction of incorporation or organization)
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(IRS Employer Identification No.) |
501 Canal Blvd, Suite A100
Richmond, California 94804
(Address of principal executive offices)
(510) 970-6000
(Registrants telephone number, including area code)
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 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 is a large accelerated filer, an accelerated filer,
or a non-accelerated filer. See definition of accelerated filer and large accelerated filer in
Rule 12b-2 of the Exchange Act.
Large accelerated filer o Accelerated filer þ Non-accelerated filer o
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
As of April 24, 2007, 35,065,236 shares of the issuers common stock, par value $0.01 per
share, were outstanding.
INDEX
SANGAMO BIOSCIENCES, INC.
Some statements contained in this report are forward-looking with respect to our operations,
research and development activities, operating results and financial condition. Statements that are
forward-looking in nature should be read with caution because they involve risks and uncertainties,
which are included, for example, in specific and general discussions about:
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our strategy; |
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product development and commercialization of our products; |
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clinical trials; |
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revenues from existing and new collaborations; |
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sufficiency of our cash resources; |
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our research and development and other expenses; |
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our operational and legal risks; and |
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our plans, objectives, expectations and intentions and any other statements that are
not historical facts. |
Various terms and expressions similar to them are intended to identify these cautionary statements.
These terms include: anticipates, believes, continues, could,
estimates, expects, intends, may, plans, seeks, should and will. Actual results may
differ materially from those expressed or implied in those statements. Factors that could cause
these differences include, but are not limited to, those discussed under Risk Factors and
Managements Discussion and Analysis of Financial Condition and Results of Operations. Sangamo
undertakes no obligation to publicly release any revisions to forward-looking statements to reflect
events or circumstances arising after the date of this report. Readers are cautioned not to place
undue reliance on the forward-looking statements, which speak only as of the date of this Quarterly
Report on Form 10-Q.
2
PART 1. FINANCIAL INFORMATION
ITEM 1. FINANCIAL STATEMENTS
SANGAMO BIOSCIENCES, INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
(In thousands, except share and per share amounts)
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March 31, |
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December 31, |
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2007 |
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2006 (1) |
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(unaudited) |
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Assets |
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Current assets: |
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Cash and cash equivalents |
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$ |
11,030 |
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$ |
12,702 |
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Marketable securities |
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37,972 |
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41,218 |
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Interest receivable |
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6 |
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55 |
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Accounts receivable |
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220 |
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487 |
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Prepaid expenses |
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519 |
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594 |
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Total current assets |
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49,747 |
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55,056 |
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Property and equipment, net |
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751 |
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675 |
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Other assets |
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49 |
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49 |
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Total assets |
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$ |
50,547 |
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$ |
55,780 |
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Liabilities and stockholders equity |
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Current liabilities: |
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Accounts payable and accrued liabilities |
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$ |
1,530 |
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$ |
1,726 |
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Accrued compensation and employee benefits |
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588 |
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878 |
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Deferred revenue |
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2,724 |
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2,596 |
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Total current liabilities |
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4,842 |
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5,200 |
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Deferred revenue, non-current portion |
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1,750 |
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1,875 |
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Total liabilities |
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6,592 |
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7,075 |
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Stockholders equity: |
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Common stock, $0.01 par value; 80,000,000
shares authorized, 35,064,032 and 35,045,398
shares issued and outstanding at March 31,
2007 and December 31, 2006, respectively |
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350 |
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350 |
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Additional paid-in capital |
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177,124 |
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176,513 |
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Accumulated deficit |
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(133,631 |
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(128,272 |
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Accumulated other comprehensive income |
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112 |
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114 |
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Total stockholders equity |
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43,955 |
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48,705 |
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Total liabilities and stockholders equity |
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$ |
50,547 |
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$ |
55,780 |
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(1) |
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Amounts derived from Audited Consolidated Financial Statements dated December 31, 2006 filed
as a part of our 2006 Form 10-K. |
See accompanying notes.
3
SANGAMO BIOSCIENCES, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(In thousands, except per share amounts)
(Unaudited)
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Three months ended |
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March 31, |
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2007 |
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2006 |
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Revenues: |
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Collaboration agreements |
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$ |
1,150 |
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$ |
1,873 |
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Research grants |
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272 |
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263 |
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Total revenues |
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1,422 |
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2,136 |
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Operating expenses: |
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Research and development |
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5,430 |
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3,589 |
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General and administrative |
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1,999 |
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1,755 |
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Total operating expenses |
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7,429 |
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5,344 |
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Loss from operations |
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(6,007 |
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(3,208 |
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Interest and other income, net |
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648 |
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464 |
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Net loss |
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$ |
(5,359 |
) |
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$ |
(2,744 |
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Basic and diluted net loss per share |
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$ |
(0.15 |
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$ |
(0.09 |
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Shares used in computing basic and diluted net loss per share |
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35,057 |
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30,600 |
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See accompanying notes.
4
SANGAMO BIOSCIENCES, INC.
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
(In thousands)
(Unaudited)
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Three months ended |
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March 31, |
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2007 |
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2006 |
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Operating Activities: |
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Net loss |
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$ |
(5,359 |
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$ |
(2,744 |
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Adjustments to reconcile net loss to net cash used in operating activities: |
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Depreciation and amortization |
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50 |
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49 |
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Amortization of (premium) / discount on investments |
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(502 |
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(25 |
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Realized loss on investments |
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(2 |
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(5 |
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Stock-based compensation |
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543 |
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451 |
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Changes in operating assets and liabilities: |
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Interest receivable |
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49 |
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18 |
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Accounts receivable |
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267 |
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679 |
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Prepaid expenses and other assets |
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75 |
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(17 |
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Accounts payable and accrued liabilities |
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(196 |
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(740 |
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Accrued compensation and employee benefits |
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(290 |
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(437 |
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Deferred revenue |
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3 |
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(1,517 |
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Net cash used in operating activities |
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(5,362 |
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(4,288 |
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Investing Activities: |
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Purchases of investments |
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(18,753 |
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(6,186 |
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Maturities of investments |
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22,501 |
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5,514 |
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Purchases of property and equipment |
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(126 |
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(214 |
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Net cash provided by / (used in) investing activities |
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3,622 |
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(886 |
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Financing Activities: |
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Proceeds from issuance of common stock |
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68 |
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65 |
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Net cash provided by financing activities |
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68 |
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65 |
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Net decrease in cash and cash equivalents |
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(1,672 |
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(5,109 |
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Cash and cash equivalents, beginning of period |
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12,702 |
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18,507 |
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Cash and cash equivalents, end of period |
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$ |
11,030 |
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$ |
13,398 |
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See accompanying notes.
5
SANGAMO BIOSCIENCES, INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS
(Unaudited) March 31, 2007
NOTE 1-BASIS OF PRESENTATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES
BASIS OF PRESENTATION
The accompanying unaudited condensed consolidated financial statements of Sangamo Biosciences,
Inc. (Sangamo or the Company) have been prepared in accordance with generally accepted
accounting principles for interim financial information and pursuant to the rules and regulations
of the Securities and Exchange Commission (SEC). Accordingly, they do not include all of the
information and footnotes required by generally accepted accounting principles for complete
financial statements. In the opinion of management, all adjustments (consisting of normal recurring
adjustments) considered necessary for a fair presentation have been included. The condensed
consolidated financial statements include the accounts of Sangamo and its wholly-owned subsidiary,
Gendaq Limited, after elimination of all material intercompany balances and transactions. Operating
results for the three months ended March 31, 2007 are not necessarily indicative of the results
that may be expected for the year ending December 31, 2007. These financial statements should be
read in conjunction with the financial statements and footnotes thereto for the year ended December
31, 2006, included in Sangamos Form 10-K as filed with the SEC.
USE OF ESTIMATES
The preparation of financial statements in conformity with generally accepted accounting
principles requires management to make estimates and assumptions that affect the amounts reported
in the financial statements and the accompanying notes. Actual results could differ from those
estimates.
FOREIGN CURRENCY TRANSLATION
The Company records foreign currency transactions at the exchange rate prevailing at the date
of the transaction. Monetary assets and liabilities denominated in foreign currency are translated
into U.S. dollars at the exchange rates in effect at the balance sheet date. All currency
translation adjustments arising from foreign currency transactions are recorded through statements
of operations.
REVENUE RECOGNITION
In accordance with Staff Accounting Bulletin No. 104, Revenue Recognition, revenue from
research activities made under strategic partnering agreements and Enabling Technology
collaborations is recognized as the services are provided when
there is persuasive evidence that an arrangement exists, delivery has occurred, the price is
fixed or determinable, and collectibility is reasonably assured. Amounts received in advance under
such agreements are deferred until the above criteria are met and the research services are
performed. Sangamos research grants are typically multi-year agreements and provide for the
reimbursement of qualified expenses for research and development as defined under the terms of the
grant agreement. Revenue under grant agreements is recognized when the related qualified research
expenses are incurred. Grant reimbursements are received on a quarterly or monthly basis and are
subject to the issuing agencys right of audit.
Milestone payments under research, partnering, or licensing agreements are recognized as
revenue upon the achievement of mutually agreed upon milestones, provided that (i) the milestone
event is substantive and its achievement is not reasonably assured at the inception of the
agreement, and (ii) there are no remaining performance obligations associated with the milestone
payment.
In accordance with Emerging Issues Task Force Issue No. 00-21, Revenue Arrangements with
Multiple Deliverables, revenue arrangements entered into after June 15, 2003, that include
multiple deliverables, are divided into separate units of accounting if the deliverables meet
certain criteria, including whether the fair value of the delivered items can be determined and
whether there is evidence of fair value of the undelivered items. In addition, the consideration is
allocated among the separate units of accounting based on their fair values, and the applicable
revenue recognition criteria are considered separately for each of the separate units of
accounting.
6
RESEARCH AND DEVELOPMENT EXPENSES
Research and development expenses consist of costs incurred for Company-sponsored as well as
collaborative research and development activities. These costs include direct and research-related
overhead expenses, which include salaries and other personnel-related expenses, stock-based
compensation, pre-clinical and clinical studies, facility costs, laboratory supplies and
depreciation of facilities and laboratory equipment, as well as the cost of funding research at
universities and other research institutions, and are expensed as incurred. Costs to acquire
technologies that are utilized in research and development and that have no alternative future use
are expensed as incurred.
STOCK-BASED COMPENSATION
Employee stock-based compensation expense recognized in first quarters of 2007 and 2006 was
calculated based on awards ultimately expected to vest and has been reduced for estimated
forfeitures. FAS 123R requires forfeitures to be estimated at the time of grant and revised, if
necessary, in subsequent periods if actual forfeitures differ from those estimates.
The following table shows total stock-based employee compensation expense included in the condensed
consolidated statement of operations for the three-month periods ended March 31, 2007 and 2006 (in
thousands):
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Three months ended |
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March 31, |
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2007 |
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2006 |
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Costs and expenses: |
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Research and development |
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$ |
343 |
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$ |
317 |
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General and administrative |
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196 |
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113 |
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Total stock-based compensation expense |
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$ |
539 |
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$ |
430 |
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There was no capitalized stock-based employee compensation cost as of March 31, 2007 and 2006.
There were no recognized tax benefits during the three months ended March 31, 2007 and 2006.
As of March 31, 2007, total compensation cost related to nonvested stock options to be recognized
in future periods was $5.54 million, which is expected to be expensed over a weighted average
period of 48 months.
Valuation Assumptions
The employee stock-based compensation expense recognized under FAS123R was determined using the
Black Scholes option valuation model. Option valuation models require the input of subjective
assumptions and these assumptions can vary over time.
Sangamo primarily bases its determination of expected volatility through its assessment of the
historical volatility of Sangamos Common Stock. The Company does not believe that it is able to
rely on its historical exercise and post-vested termination activity to provide accurate data for
estimating its expected term for use in determining the fair value of these options. Therefore, as
allowed by Staff Accounting Bulletin (SAB) No. 107, Share-Based Payment , the Company have opted to
use the simplified method for estimating its expected term equal to the midpoint between the
vesting period and the contractual term.
7
The weightedaverage assumptions used for estimating the fair value of the employee stock options
are as follows:
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Three months ended |
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March 31, |
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2007 |
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2006 |
Risk-free interest rate |
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4.58 |
% |
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4.94 |
% |
Expected life of option |
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6.25 years |
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6.25 years |
Expected dividend yield of stock |
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0.0 |
% |
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0.0 |
% |
Expected volatility |
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0.93 |
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0.97 |
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The weightedaverage assumptions used for estimating the fair value of the employees purchase
rights are as follows:
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Three months ended |
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March 31, |
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2007 |
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2006 |
Risk-free interest rate |
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3.6.-5.1 |
% |
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3.17-4.26 |
% |
Expected life of option |
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0.5-2 years |
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0.5-2 yrs |
Expected dividend yield of stock |
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0.0 |
% |
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0.0 |
% |
Expected volatility |
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0.46-0.77 |
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0.41-0.64 |
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8
Stock Option Activity
A summary of Sangamos stock option activity follows:
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Options Outstanding |
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Weighted |
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Average |
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Shares Available |
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Weighted-Average |
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Remaining |
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for Grant of |
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Number of |
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Exercise per |
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Contractual |
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Options |
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Shares |
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Share Price |
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Term |
|
Balance at January 1, 2007 |
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|
3,715,021 |
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|
4,067,812 |
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|
$ |
5.65 |
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Options granted |
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|
(210,750 |
) |
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|
210,750 |
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|
$ |
6.91 |
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Options exercised |
|
|
|
|
|
|
(18,634 |
) |
|
$ |
3.65 |
|
|
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|
|
Options canceled |
|
|
21,718 |
|
|
|
(21,718 |
) |
|
$ |
7.74 |
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Balance at March 31, 2007 |
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|
3,525,989 |
|
|
|
4,238,210 |
|
|
$ |
5.71 |
|
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|
6.35 |
|
|
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|
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|
Options exercisable at March 31, 2007 |
|
|
|
|
|
|
2,611,416 |
|
|
$ |
5.76 |
|
|
|
4.86 |
|
There were no shares subject to Sangamos right of repurchase as of March 31, 2007. The intrinsic
value of options exercised were $81,000 and $794,000 for the three months ended March 31, 2007 and
2006, respectively.
The weighted-average estimated fair value per share of options granted during the three months
ended March 31, 2007 and 2006 were $5.45 and $4.13, respectively, based upon the assumptions in the
Black-Scholes valuation model described above.
The weighted-average estimated fair value per share of employee purchase rights during the three
months ended March 31, 2007 and 2006 were $2.17 and $1.70, respectively, based upon the assumptions
in the Black-Scholes valuation model described above.
The following table summarizes information with respect to stock options outstanding at March 31,
2007:
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|
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Options Outstanding |
|
|
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|
|
|
Weighted Average |
|
|
|
|
|
|
Remaining |
Range of Exercise Price |
|
Number of Shares |
|
Contractual Life |
|
|
|
|
(In Years) |
$ 0.05 $0.17
|
|
|
461,583 |
|
|
|
1.07 |
|
$ 0.23 $3.80
|
|
|
433,362 |
|
|
|
6.57 |
|
$ 3.87 $4.11
|
|
|
647,425 |
|
|
|
7.83 |
|
$ 4.15 $5.18
|
|
|
231,325 |
|
|
|
7.22 |
|
$ 5.19 $5.19
|
|
|
475,315 |
|
|
|
7.04 |
|
$ 5.30 $6.77
|
|
|
255,250 |
|
|
|
7.19 |
|
$ 6.82 $6.82
|
|
|
475,000 |
|
|
|
9.70 |
|
$ 6.92 $7.49
|
|
|
646,750 |
|
|
|
6.22 |
|
$
7.56 $14.60
|
|
|
509,200 |
|
|
|
5.13 |
|
$14.87 $38.00
|
|
|
103,000 |
|
|
|
3.86 |
|
|
|
|
|
|
|
|
|
|
|
|
|
4,238,210 |
|
|
|
6.35 |
|
|
|
|
|
|
|
|
|
|
At March 31, 2007, the aggregate intrinsic values of the outstanding and exercisable options were
$7.9 million and $5.8 million, respectively.
Sangamo did not grant any stock option to consultants during the three months ended March 31, 2007
and 2006. The Company granted 10,000 nonqualified stock options in July 2006. The options
generally vest over four years at a rate of 25 percent one year from grant date and
one-thirty-sixth per month thereafter and expire ten years after the grant date. The fair value of
these options was determined using the Black-Scholes Merton model. Total nonqualified stock-based
compensation expense was $3,000 and $4,700 for the three months period ended March 31, 2007 and
2006, respectively.
RECENT ACCOUNTING PRONOUNCEMENT
In February 2007, the Financial Accounting Standards Board (FASB) issued Statement of Financial
Accounting Standards (SFAS) No. 159, The Fair Value Option for Financial Assets and Financial
Liabilities, Including an Amendment of FASB Statement No. 115, which will become effective in
2008. SFAS No. 159 permits entities to measure eligible financial assets, financial liabilities and
firm commitments at fair value, on an instrument-by-instrument basis, that are otherwise not
permitted to be accounted for at fair
9
value under other generally accepted accounting principles.
The fair value measurement election is irrevocable and subsequent changes in fair value must be
recorded in earnings. The Company is evaluating what impact, if any; the adoption of this standard
will have on its financial position or results of operations.
In September 2006 the FASB issued FASB Statement No. 157, Fair Value Measurements, or SFAS 157.
The standard provides guidance for using fair value to measure assets and liabilities. The
standard also responds to investors requests for expanded information about the extent to which
companies measure assets and liabilities at fair value, the information used to measure fair value,
and the effect of fair value measurements on earnings. The standard applies whenever other
standards require or permit assets or liabilities to be measured at fair value. The standard does
not expand the use of fair value in any new circumstances. SFAS 157 must be adopted prospectively
as of the beginning of the year it is initially applied. SFAS 157 is effective for financial
statements issued for fiscal years beginning after November 15, 2007, and interim periods within
those fiscal years. The Company is evaluating what impact, if any; the adoption of this standard
will have on its financial position or results of operations.
NOTE 2-BASIC AND DILUTED NET LOSS PER SHARE
Basic earnings (loss) per share is calculated based on the weighted average number of shares
of common stock outstanding during the period. Diluted earnings (loss) per share is calculated
based on the weighted average number of shares of common stock and dilutive securities, such as
stock options and equivalents, outstanding during the period. Potential dilutive shares of common
stock resulting from the assumed exercise of outstanding stock options and equivalents.
Because Sangamo is in a net loss position, diluted earnings (loss) per share excludes the
effects of common stock equivalents consisting of options, which are all antidilutive. Had Sangamo
been in a net income position, diluted earnings (loss) per share would have included the shares
used in the computation of basic net loss per share as well as an additional 1,622,716 shares and
1,522,496 shares for the three months ended 2007 and 2006, respectively, related to outstanding
options.
NOTE 3-COMPREHENSIVE LOSS
Comprehensive loss is comprised of net loss and other comprehensive income (loss). Other
comprehensive income (loss) includes certain changes in stockholders equity that are excluded from
net loss, which includes unrealized gains and losses on available-for-sale securities.
Comprehensive loss and its components are as follows (in thousands):
|
|
|
|
|
|
|
|
|
|
|
Three months ended |
|
|
|
March 31, |
|
|
|
2007 |
|
|
2006 |
|
Net loss |
|
$ |
(5,359 |
) |
|
$ |
(2,744 |
) |
Changes in unrealized gain on securities available-for-sale |
|
|
(2 |
) |
|
|
(11 |
) |
|
|
|
|
|
|
|
Comprehensive loss |
|
$ |
(5,361 |
) |
|
$ |
(2,755 |
) |
|
|
|
|
|
|
|
NOTE 4-MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES
Recently Terminated Strategic Partnership with Edwards Lifesciences
In December 2006, Sangamo entered into an Asset Purchase Agreement with Edwards Lifesciences LLC
(Edwards) to acquire all of the assets in Edwards ZFP TF angiogenesis program, including
regulatory filings, clinical data, and GMP product in exchange for one million shares of our
unregistered Common Stock and certain royalties. This transaction was valued at $5.8 million based
on the fair value of our publicly traded stock at the closing date of the transaction less a
discount for lack of marketability in the unregistered Common Stock. Under the agreement, Company
agreed to pay Edwards royalties generated by the sales of certain human therapeutic products,
including products to treat ischemic cardiovascular and vascular disease and diabetic neuropathy,
based upon ZFP TF activation of the VEGF gene: the first product is not expected to be available
for sale before 2012. The amount of royalties payable to Edwards is equal to (i) five percent (5%)
of the net sales of each such product sold by Sangamo and (ii) the greater of (a) five percent (5%)
of the net sales of each such product sold by a sublicensee of Sangamo or (b) twenty-five percent
(25%) of the royalty payment received by Sangamo from its sublicensee on account of such product
sold by such sublicensee; provided that total royalties paid by Sangamo under the agreement shall
not exceed $20 million in any calendar year or $100 million in the aggregate. In connection with
this transaction, the Company and Edwards terminated their prior agreements entered in January
2000.
Enabling Technology Collaborations for Pharmaceutical Protein Production
10
We have established several research collaborations in this area. In December 2004, we
announced a research collaboration agreement with Pfizer Inc to use our ZFP technology to develop
enhanced cell lines for protein pharmaceutical production. The scope of this agreement was expanded
in December 2006 and provided further research funding from Pfizer to develop additional cell lines
for enhanced protein production. Under the terms of the agreement, Pfizer is funding research at
Sangamo and Sangamo will provide our proprietary ZFP technology for Pfizer to assess its
feasibility for use in mammalian cell-based protein production. We are generating novel cell lines
and vector systems for enhanced protein production as well as novel technology for rapid creation
of new production cell lines. Revenues attributable to collaborative research and development
performed under the Pfizer agreement were $25,000 and $150,000 during the three months ended March
31, 2007 and 2006, respectively. Related research and development costs and expenses performed
under the Pfizer agreement were $134,000 and $57,000 during the three months ended March 31, 2007
and 2006, respectively.
Plant Agriculture Agreements
Sangamo scientists and collaborators have shown that ZFP TFs and ZFP nucleases (ZFNs) can be
used to regulate and modify genes in plants with similar efficacy to that shown in various
mammalian cells and organisms. The ability to regulate gene expression with engineered ZFP TFs may
lead to the creation of new plants that increase crop yields, lower production costs, are more
resistant to herbicides, pesticides, and plant pathogens; and permit the development of branded
agricultural products with unique nutritional and processing characteristics. In addition, ZFNs may
be used to facilitate the efficient and reproducible generation of transgenic plants. Effective as
of October 1, 2005, we entered into a Research License and Commercial Option Agreement with Dow
AgroSciences LLC (DAS), a wholly owned indirect subsidiary of Dow Chemical Corporation. Under
this agreement, we will provide DAS with access to our proprietary ZFP technology and the exclusive
right to use our ZFP technology to modify the genomes or alter the nucleic acid or protein
expression of plant cells, plants, or plant cell cultures. We will retain rights to use plants or
plant-derived products to deliver ZFP TFs or ZFNs into human or animals for diagnostic,
therapeutic, or prophylactic purposes.
Our agreement with DAS provides for an initial three-year research term during which time we
will work together to validate and optimize the application of our ZFP technology to plants, plant
cells and plant cell cultures. A joint committee having equal representation from both companies
will oversee this research. During the initial three-year research term, DAS will have the option
to obtain a commercial license to sell products incorporating or derived from plant cells generated
using our ZFP technology, including agricultural crops, industrial products and plant-derived
biopharmaceuticals. This commercial license will be exclusive for all such products other than
animal and human health products. In the event that DAS exercises this option, DAS may elect to
extend the research program beyond the initial three-year term on a year-to-year basis.
Pursuant to the Research License and Commercial Option Agreement, DAS made an initial cash
payment to us of $7.5 million and agreed to purchase up to $4.0 million of our
common stock in the next financing transaction meeting certain criteria. In November 2005, the
Company sold approximately 1.0 million shares of common stock to DAS at a price of $3.85 per share,
resulting in gross proceeds of $3.9 million. In addition, DAS will provide between $4.0 and $6.0
million in research funding over the initial three-year research term and may make an additional
payment of up to $4.0 million in research milestone payments to us during this same period,
depending on the success of the research program. In the event that DAS elects to extend the
research program beyond the initial three-year term, DAS will provide additional research funding.
If DAS exercises its option to obtain a commercial license, we will be entitled to full payment of
the $4.0 million in research milestones, a one-time exercise fee of $6.0 million, minimum annual
payments of up to $25.25 million, development and commercialization milestone payments for each
product, and royalties on sales of products. Furthermore, DAS will have the right to sublicense our
ZFP technology to third parties for use in plant cells, plants, or plant cell cultures, and we will
be entitled to 25% of any cash consideration received by DAS under such sublicenses.
We have agreed to supply DAS and its sublicensees with ZFP TFs and/or ZFNs for both research
and commercial use. If DAS exercises its option to obtain a commercial license, DAS may request
that we transfer, at DASs expense, the ZFP manufacturing technology to DAS or to a mutually
agreed-upon contract manufacturer.
The Research License and Commercial Option Agreement will terminate automatically if DAS fails
to exercise its option for a commercial license by the end of the initial three-year research term.
DAS may also terminate the agreement at the end of the second year of the initial research term if
the joint committee overseeing the research determines that disappointing research results have
made it unlikely that DAS will exercise the option; we are guaranteed to receive $4.0 million in
research funding from DAS prior to such a termination. Following DASs exercise of the option and
payment of the exercise fee, DAS may terminate the agreement at any time. In addition, each party
may terminate the agreement upon an uncured material breach of the other party. In the event of any
termination of the agreement, all rights to use our ZFP technology will revert to us, and DAS will
no longer be permitted to practice our ZFP technology or to develop or, except in limited
circumstances, commercialize any products derived from our ZFP technology.
11
Revenues related to the
research license under the DAS agreement are being recognized ratably over the initial three-year
research term of the agreement and were $625,000 during both the three months ended March 31, 2007
and 2006. Revenues attributable to collaborative research and development performed under the DAS
agreement were $500,000 and $948,070 during the three months ended March 31, 2007 and 2006,
respectively. Related costs and expenses incurred under the DAS agreement were $500,000 and
$862,500 during the three months ended March 31, 2007 and 2006, respectively.
Funding from Research Foundations
The Juvenile Diabetes Research Foundation International
On October 26, 2006, we announced a partnership with the Juvenile Diabetes Research Foundation
International (JDRF) to provide financial support of Sangamos upcoming Phase 2 human clinical
studies of SB-509, a ZFP Therapeutic that is in development for the treatment of diabetic
neuropathy. Under the agreement with JDRF and subject to its terms and conditions, including the
Companys achievement of certain milestones associated with the Companys Phase 2 clinical trial of
SB-509 for the treatment of diabetic neuropathy, JDRF will pay the Company an aggregate amount up
to $3.0 million. After the first commercial launch of SB-509 in a major market, JDRF has the right
to receive, subject to certain limitations, annual payments from us, until such time when the total
amount paid to JDRF, including payments made on account of the our licensing arrangements, equals
three times the amount received by us from JDRF. We are obligated to cover all costs of the Phase
2 trial that are not covered by JDRFs grant. During the quarter ended March 31, 2007, the Company
received $500,000 from JDRF upon achievement of a milestone.
The Michael J. Fox Foundation
On January 23, 2007, Sangamo announced a partnership with the Michael J. Fox Foundation (MJFF) to
provide financial support of Sangamos ZFP TFsTM to activate the expression of glial cell
line-derived neurotrophic factor (GDNF) that has shown promise in preclinical testing to slow or
stop the progression of Parkinsons disease. Under the agreement with MJFF and subject to its
terms and conditions, MJFF will pay the Company $950,000 award over a period of two years.
Revenues attributable to research and development performed under the MJFF partnership were $50,000
during the three months ended March 31, 2007.
NOTE 5-STOCKHOLDERS EQUITY
In June 2006, in an underwritten public offering and pursuant to an effective registration
statement, Sangamo sold 3,100,000 shares of common stock at a public offering price of $6.75 per
share, resulting in net proceeds of approximately $20.15 million after deducting underwriters
discount.
NOTE 6-INCOME TAXES
On January 1, 2007, the Company adopted the provisions of Financial Standards Accounting Board
Interpretation No. 48, Accounting for Uncertainty in Income Taxes-an interpretation of FASB
Statement No. 109 (FIN 48). There was no impact on the Companys financial statements upon
adoption. Because of the Companys historical significant net operating losses, it has not been
subject to income tax since inception. There were no unrecognized tax benefits during all the
periods presented.
We maintain deferred tax assets that reflect the net tax effects of temporary differences
between the carrying amounts of assets and liabilities for financial reporting purposes and the
amounts used for income tax purposes. These deferred tax assets include net operating loss
carryforwards, research credits and capitalized research and
development. The net deferred tax asset has been fully offset by a valuation allowance because
of the Companys history of losses. Utilization of operating losses and credits may be subject to
substantial annual limitation due to ownership change provisions of the Internal Revenue Code of
1986, as amended and similar state provisions. The annual limitation may result in the expiration of net
operating losses and credits before utilization.
NOTE 7-SUBSEQUENT EVENT
On April 27, 2007, we announced a partnership with the Genentech, Inc. to provide financial support
of Sangamo to develop, validate and optimize ZFNs capable of making certain targeted modifications
to the genome of Genentech cell lines. Upon successful development of such ZFNs, Sangamo will
transfer these ZFNs and the modified cell lines to Genentech and will provide technical support to
Genentech with respect to the use of the transferred ZFN technology. In consideration for the
rights and licenses granted to Genentech, as well as Sangamos development efforts, Genentech will
pay Sangamo an upfront fee, an ongoing technology access fee, and certain payments upon achievement
of specified milestones relating to the research of ZFNs and the development and commercialization
of products manufactured using a modified cell line created by ZFN technology or other technology
covered by Sangamos intellectual property rights.
12
ITEM 2. MANAGEMENTS DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
The discussion in Managements Discussion and Analysis of Financial Condition and Results of
Operations contains trend analysis, estimates and other forward-looking statements within the
meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities
Exchange Act of 1934, as amended. These forward-looking statements include, without limitation,
statements containing the words believes, anticipates, expects, continue, and other words
of similar import or the negative of those terms or expressions. Such forward-looking statements
are subject to known and unknown risks, uncertainties, estimates and other factors that may cause
the actual results, performance or achievements of the Company, or industry results, to be
materially different from any future results, performance or achievements expressed or implied by
such forward-looking statements. Actual results could differ materially from those set forth in
such forward-looking statements as a result of, but not limited to, the Risk Factors described
below. You should read the following discussion and analysis along with the financial statements
and notes attached to those statements included elsewhere in this report and in our annual report
on Form 10-K for the year ended December 31, 2006 as filed with the Securities and Exchange
Commission on March 1, 2007.
Overview
We were incorporated in June 1995. From our inception through March 31, 2007, our activities
related primarily to establishing and operating a biotechnology research and development
organization and developing relationships with our corporate collaborators. Our scientific and
business development endeavors currently focus on the engineering of novel zinc finger DNA binding
proteins (ZFPs) for the regulation and modification of genes. We have incurred net losses since
inception and expect to incur losses in the future as we continue our research and development
activities. To date, we have funded our operations primarily through the issuance of equity
securities, borrowings, payments from federal government research grants and from corporate
collaborators and strategic partners. As of March 31, 2007, we had an accumulated deficit of
$133.6 million.
Our revenues have consisted primarily of revenues from our corporate partners for ZFP TFs and
ZFNs, contractual payments from strategic partners for research programs and research milestones,
and Federal government research grant funding. We expect revenues will continue to fluctuate from
period to period and there can be no assurance that new collaborations or partner fundings will
continue beyond their initial terms.
Commencing in 2005, we have placed more emphasis on higher-value therapeutic product
development and related strategic partnerships and less emphasis on our Enabling Technology
collaborations. We believe this shift in emphasis has the potential to increase the return on
investment to our stockholders by allocating capital resources to higher value, therapeutic product
development activities. At the same time, it may reduce
our revenues over the next several years and it increases our financial risk by increasing
expenses associated with product development. We have filed an Investigational New Drug (IND)
application with the U.S. Food and Drug Administration (FDA) and have initiated a Phase 1 clinical
trial of a ZFP Therapeutic in patients with diabetic neuropathy during the first quarter of 2005.
Development of novel therapeutic products is costly and is subject to a lengthy and uncertain
regulatory process by the FDA. Our future products are gene-based therapeutics. Adverse events in
both our own clinical program and other programs in gene therapy may have a negative impact on
regulatory approval, the willingness of potential commercial partners to enter into agreements and
the perception of the public.
Research and development expenses consist primarily of salaries and related personnel
expenses, including stock-based compensation, pre-clinical and clinical studies, allocated
facilities costs, laboratory supplies, and depreciation of facilities and laboratory equipment,
subcontracted research expenses, trademark registration and technology licenses. Research and
development costs incurred in connection with collaborator-funded activities are expensed as
incurred. Costs to acquire technologies that are utilized in research and development and that have
no alternative future use are expensed as incurred. We believe that continued investment in
research and development is critical to attaining our strategic objectives. We expect these
expenses will increase significantly as we increase our focus on development of ZFP Therapeutics.
The Company is also developing ZFNs for therapeutic gene correction and therapeutic gene
modification as a treatment for certain monogenic and infectious diseases. Additionally, in order
to develop ZFP TFs and ZFNs as commercially relevant therapeutics, we expect to expend additional
resources for expertise in the manufacturing, regulatory affairs and clinical research aspects of
biotherapeutic development.
General and administrative expenses consist primarily of salaries and related personnel
expenses for executive, finance and administrative personnel, stock-based compensation,
professional fees, patent prosecution expenses, allocated facilities costs and other general
corporate expenses. As we pursue commercial development of our therapeutic leads we expect the
business aspects of the
13
Company to become more complex. We may be required in the future to add
personnel and incur additional costs related to the maturity of our business.
Critical Accounting Estimates
The preparation of financial statements in conformity with accounting principles generally
accepted in the United States requires management to make estimates and assumptions that affect the
reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at
the date of the financial statements and the reported amounts of revenues and expenses during the
reporting period. Such estimates are described in Note 1, Basis of Presentation and Summary of
Significant Accounting Policies to the Unaudited Notes to Condensed Financial Statements. We base
our estimates on historical experience and on various other assumptions that we believe to be
reasonable under the circumstances, the results of which form our basis for making
judgments about the carrying value of assets and liabilities that are not readily apparent
from other sources, and evaluates our estimates on an ongoing basis. Actual results could differ
from those estimates under different assumptions or conditions. Sangamo believes the following
critical accounting policies have significant effect in the preparation of our consolidated
financial statements.
Revenue Recognition
In accordance with Staff Accounting Bulletin No. 104, Revenue Recognition, revenue from
research activities made under strategic partnering collaborations is recognized as the services
are provided when there is persuasive evidence that an arrangement exists, delivery has occurred,
the price is fixed or determinable, and collectibility is reasonably assured. Amounts received
under such agreements are deferred until the above criteria are met and the research services are
performed. Sangamos federal government research grants are typically multi-year agreements and
provide for the reimbursement of qualified expenses for research and development as defined under
the terms of the grant agreement. Revenue under grant agreements is recognized when the related
research expenses are incurred. Grant reimbursements are typically received on a quarterly basis
and are subject to the issuing agencys right of audit.
Sangamo recognizes revenue from its Therapeutic and Enabling Technology collaborations when
ZFP-based products are delivered to the collaborators, persuasive evidence of an agreement exists,
there are no unfulfilled obligations, the price is fixed and determinable, and collectibility is
reasonably assured. Generally, Sangamo receives partial payments from these collaborations prior to
the delivery of ZFP-based products and the recognition of these revenues is deferred until the
ZFP-based products are delivered, the risk of ownership has passed to the collaborator and all
performance obligations have been satisfied. Upfront or signature payments received upon the
signing of an Enabling Technology agreement are generally recognized ratably over the applicable
period of the agreement, which currently ranges between 12 and 15 months, or as ZFP-based products
are delivered.
Milestone payments under research, partnering, or licensing agreements are recognized as
revenue upon the achievement of mutually agreed upon milestones, provided that (i) the milestone
event is substantive and its achievement is not reasonably assured at the inception of the
agreement, and (ii) there are no further significant performance obligations associated with the
milestone payment.
In accordance with Emerging Issues Task Force Issue No. 00-21, Revenue Arrangements with
Multiple Deliverables, revenue arrangements entered into after June 15, 2003, that include
multiple deliverables, are divided into separate units of accounting if the deliverables meet
certain criteria, including whether the fair value of the delivered items can be determined and
whether there is evidence of fair value of the undelivered items. In addition, the consideration is
allocated among the separate units of accounting
based on their fair values, and the applicable revenue recognition criterion is considered
separately for each of the separate units of accounting.
Stock-Based Compensation
On January 1, 2006, we began accounting for employee stock-based compensation in accordance with
FAS 123R. Under the provisions of FAS 123R, employee stock-based compensation is estimated at the
date of grant based on the employee stock awards fair value using the Black-Scholes option-pricing
model and is recognized as expense ratably over the requisite service period in a manner similar to
other forms of compensation paid to employees. The Black-Scholes option-pricing model requires the
use of certain subjective assumptions. The most significant of these assumptions are our estimates
of the expected volatility of the market price of our stock and the expected term of the award. We
primarily base our determination of expected volatility through our assessment of the historical
volatility of our Common Stock. We do not believe that we are able to rely on our historical
exercise and post-vested termination activity to provide accurate data for estimating our expected
term for use in determining the fair value of these options. Therefore, as allowed by Staff
Accounting Bulletin (SAB) No. 107, Share-Based Payment , we have opted to use the
simplified method
for estimating our expected term equal to the midpoint between the vesting period and the
contractual term. As required under the accounting rules, we review our valuation assumptions at
each grant date and, as a result, our valuation assumptions used to value employee stock-based
awards granted in future periods may change.
14
RESULTS OF OPERATIONS
Three months ended March 31, 2007 and 2006
Revenues
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three months ended March 31, |
|
|
|
(in thousands, except percentage values) |
|
|
|
2007 |
|
|
2006 |
|
|
Change |
|
|
% |
|
Revenues: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Collaboration agreements |
|
$ |
1,150 |
|
|
$ |
1,873 |
|
|
$ |
(723 |
) |
|
|
(39 |
%) |
Research grants |
|
|
272 |
|
|
|
263 |
|
|
|
9 |
|
|
|
3 |
% |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total revenues |
|
$ |
1,422 |
|
|
$ |
2,136 |
|
|
$ |
(714 |
) |
|
|
(33 |
%) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
We are increasing the emphasis of our research and development activities on ZFP Therapeutics
and are moving away from our historic emphasis on Enabling Technology agreements. Over the next
several years, this change in resource allocation will reduce our revenues.
Total revenues decreased to $1.4 million for the three months ended March 31, 2007 from $2.1
million in the corresponding period in 2006. The decrease for the three months ended March 31, 2007
was principally due to decrease revenues in connection with our Research License and Commercial
Option Agreement with Dow AgroSciences LLC (DAS), a wholly owned indirect subsidiary of Dow
Chemical Corporation, of $448,000, Johnson & Johnson of $150,000 and Pfizer of $125,000. We
anticipate continued revenues from collaboration agreements through the end of 2007, and we have
applied for, and plan to continue to apply for, federal government research grants in the future to
support the development of applications of our technology platform. Although we have negotiated
collaboration agreements and received federal government research grants in the past, we cannot
assure you that these efforts will be successful in the future.
Operating Expenses
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three months ended March 31, |
|
|
|
(in thousands, except percentage values) |
|
|
|
2007 |
|
|
2006 |
|
|
Change |
|
|
% |
|
Operating Expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
5,430 |
|
|
$ |
3,589 |
|
|
$ |
1,841 |
|
|
|
51 |
% |
General and administrative |
|
|
1,999 |
|
|
|
1,755 |
|
|
|
244 |
|
|
|
14 |
% |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total expenses |
|
$ |
7,429 |
|
|
$ |
5,344 |
|
|
$ |
2,085 |
|
|
|
39 |
% |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development
Over the past three fiscal years, research and development expenses have consisted primarily
of salaries and related personnel expenses including stock-based compensation as well as laboratory
supplies, allocated facilities costs, subcontracted research expenses,
trademark registration and technology licenses. We expect to continue to devote substantial
resources to research and development in the future and expect research and development expenses to
increase in the next several years if we are successful in advancing our ZFP Therapeutic product
candidates into clinical trials. To the extent we collaborate with others with respect to clinical
trials, increases in research and development expenses may be reduced or avoided.
Research and development expenses for the first quarter of 2007 increased to $7.4 million
compared to $5.3 million for the first quarter of 2006. The increase in research and development
expenses for the three months ended March 31, 2007 was primarily attributable to increased external
development expenses of $1.3 million, primarily associated with our diabetic neuropathy program,
increased personnel and lab supply expenses of $374,000 and $246,000, respectively, due to
increased headcount. Stock-based compensation was comparable for three months ended March 31, 2007
and 2006.
General and administrative
15
General and administrative expenses consist primarily of salaries and related personnel
expenses for executive, finance and administrative personnel, stock-based compensation,
professional fees, patent prosecution expenses, allocated facilities costs, other general corporate
expenses and stock-based compensation. As we pursue commercial development of our therapeutic
leads, we expect the business aspects of the Company to become more complex. We may be required in
the future to add personnel and incur additional costs related to the maturity of our business.
General and administrative expenses were $2.0 million in the three months ended March 31,
2007, as compared to $1.8 million during the corresponding period in 2006. This increase is
primarily related to increased patent prosecution and corporate legal expenses of $126,000 and $81,000,
respectively. Stock-based compensation was comparable for three months ended March 31, 2007 and
2006.
Interest and Other Income, net
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three months ended March 31, |
|
|
|
(in thousands, except percentage values) |
|
|
|
2007 |
|
|
2006 |
|
|
Change |
|
|
% |
|
Interest and other income, net |
|
$ |
648 |
|
|
$ |
464 |
|
|
$ |
184 |
|
|
|
40 |
% |
Interest and other income, net, increased to $648,000 for the three months ended March 31,
2007 from $464,000 in the corresponding period in 2006. The increase was primarily related to
higher interest income of $212,000 related to higher average investment balances during the quarter
ended March 31, 2007 from the June 2006 equity financing. In addition, a foreign currency
translation gain of $7,000 was recorded during the quarter ended March 31, 2007 versus a foreign
currency translation gain of $35,000 during the corresponding quarter in 2006.
Liquidity and Capital Resources
Since inception, we have financed our operations primarily through the sale of equity
securities, payments from corporate collaborators, federal government research grants and financing
activities such as a bank line of credit. As of March 31, 2007, we had cash, cash equivalents,
investments and interest receivable totaling $49.0 million.
Net cash used in operating activities was $5.4 million for the three months ended March 31,
2007. Net cash used consisted of the net loss for the three-month period of $5.4 million.
Net cash used for operating activities was $4.3 million for the three months ended March 31, 2006.
Net cash used consisted primarily of the net loss for the three month period of $2.7 million and a
net change of $2.0 million in operating assets and liabilities. This was partially offset by
stock-based compensation charges of $451,000 and depreciation and amortization of $49,000.
Net cash provided by investing activities was $3.6 million for the three months ended March
31, 2007 and was primarily due to cash proceeds associated with maturities of investments of $22.5
million partially offset by cash used to purchase investments and fixed assets of $18.8 million and
$126,000, respectively. Net cash used in investing activities was $886,000 for the three months
ended March 31, 2006 and was primarily comprised of cash used to purchase investments and fixed
assets of $6.2 million and 214,000, respectively partially offset by proceeds associated with
maturities of investments of $5.5 million.
Net cash provided by financing activities for the three-month period ended March 31, 2007 and
2006 was $68,000 and $65,000, respectively. Proceeds from both years were solely related to
proceeds from issuance of common stock related to stock options exercises.
While we expect our rate of cash usage to increase in the future, in particular, in support of
our product development endeavors, we believe that the available cash resources, funds received
from corporate collaborators, strategic partners and federal government research grants will be
sufficient to finance our operations through 2008. We may need to raise additional capital to fund
our ZFP Therapeutic development activities. Additional capital may not be available in terms
acceptable to us, or at all. If adequate funds are not available, our business and our ability to
develop our technology and our ZFP Therapeutic products would be harmed.
ITEM 3. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK
Our exposure to market risk for changes in interest rates relates primarily to our cash equivalents
and investments. The investments are available-for-sale. We do not use derivative financial
instruments in our investment portfolio. We attempt to ensure the safety and preservation of our
invested funds by limiting default and market risks. Our cash and investments policy emphasizes
liquidity and preservation of principal over other portfolio considerations. We select investments
that maximize interest income to the extent
16
possible within these guidelines. We satisfy liquidity
requirements by investing
excess cash in securities with different maturities to match projected cash needs and limit
concentration of credit risk by diversifying our investments among a variety of high credit-quality
issuers. We mitigate default risk by investing in only investment-grade securities. The portfolio
includes marketable securities with active secondary or resale markets to ensure portfolio
liquidity. All investments have a fixed interest rate and are carried at market value, which
approximates cost.
Our market risks at March 31, 2007 have not changed materially from those discussed in Item 7A of
our Form 10-K for the year ended December 31, 2006 on file with the Securities and Exchange
Commission.
ITEM 4. CONTROLS AND PROCEDURES
(a) Evaluation of Disclosure Controls and Procedures
The Companys management, with the participation of the Companys Chief Executive Officer and
Principal Financial Officer, evaluated the effectiveness of the Companys disclosure controls and
procedures (as defined in Exchange Act Rule 13a-15(e) or 15d-15(e)) as of the end of the period
covered by this report. Based on that evaluation, the Chief Executive Officer and Principal
Financial Officer concluded that the Companys disclosure controls and procedures as of the end of
the period covered by this report are functioning effectively to provide reasonable assurance that
the information required to be disclosed by the Company in reports filed under the Securities
Exchange Act of 1934 is recorded, processed, summarized and reported within the time periods
specified in the SECs rules and forms, and that such information is accumulated and communicated
to management, including our Chief Executive Officer and Principal Financial Officer, or persons
performing similar functions, as appropriate to allow timely decisions regarding required
disclosure.
(b) Change in Internal Control over Financial Reporting
No change in the Companys internal control over financial reporting occurred during the
Companys most recent fiscal quarter that has materially affected, or is reasonably likely to
materially affect, the Companys internal control over financial reporting.
17
PART II. OTHER INFORMATION
ITEM 1. LEGAL PROCEEDINGS
We are not party to any material pending legal proceedings, other than routine litigation
incidental to our business.
ITEM 1A. RISKS FACTORS
This Form 10-Q contains forward-looking information based on our current expectations. Because our
actual results may differ materially from any forward-looking statements made by or on behalf of
Sangamo, this section includes a discussion of important factors that could affect our actual
future results, including, but not limited to, our revenues, expenses, net loss and loss per share.
We have increased the focus of our research and development programs on human therapeutics,
which will increase operating expenditures and the uncertainty of our business. We are increasing
the emphasis and focus of our research and development activities on ZFP Therapeutics and have
fewer resources invested in our Enabling Technology programs. In the short term, this change may
reduce our revenues and increase operating expenditures due to larger financial outlays to fund
preclinical studies, manufacturing, and clinical research. The focus on ZFP Therapeutics will also
increase the visibility of our lead therapeutic programs and the potential impact on the stock
price of news releases relating to these programs.
We are conducting proprietary research to discover ZFP Therapeutic product candidates. These
programs increase our financial risk of product failure, may significantly increase our research
expenditures, and may involve conflicts with our collaborators and strategic partners. Our
proprietary research programs consist of research which is funded solely by the Company and where
the Company retains exclusive rights to therapeutic products generated by the research. This is in
contrast to certain of our research programs that may be funded by corporate partners and in which
we may share rights to any resulting products. We have conducted proprietary research since
inception, however, in the past several years, our strategy has shifted toward placing greater
emphasis on proprietary research and therapeutic development and we expect this trend will continue
in 2007 as we prosecute our first Phase 2 clinical trial and bring new ZFP Therapeutics into
clinical trials. Conducting proprietary research programs may not generate corresponding revenue
and may create conflicts with our collaborators or strategic partners. The implementation of this
strategy will involve substantially greater business risks, the expenditure of significantly
greater funds than our historic research activities and will require substantial commitments of
time from our management and staff.
In addition, disagreements with our collaborators or strategic partners could develop over
rights to our intellectual property with respect to our proprietary research activities. Any
conflict with our collaborators or strategic partners could reduce our ability to enter into future
collaboration or strategic partnering agreements and negatively impact our relationship with
existing collaborators and strategic partners, which could reduce our revenue and delay or
terminate our product development.
We have initiated several Phase 1 clinical trials in our lead ZFP Therapeutic program, and ZFP
Therapeutics have undergone limited testing in humans. We have completed enrollment and treatment
of the patients in the first of these trials of SB-509 for diabetic neuropathy and thus far have
not observed any serious drug-related adverse events. However if our lead ZFP Therapeutic fails one
of its initial safety studies, it could reduce our ability to attract new investors and corporate partners. In January 2005, we filed
an IND with the FDA for SB-509, a ZFP TF activator of VEGF-A, for the treatment of mild to moderate
diabetic neuropathy. We have completed enrollment and treatment of a Phase 1, single blind,
dose-escalation trial to measure the laboratory and clinical safety of SB-509 and initiated a Phase
2 clinical trial for this indication. In addition, Phase 1 clinical trials of an identical ZFP TF
has been carried out in subjects with peripheral artery disease. These early studies of a ZFP
Therapeutic are a highly visible test of our ZFP Therapeutic approach. Since we have increased our
focus on ZFP Therapeutic research and development, investors will increasingly assess the value of
our technology based on the continued progress of ZFP Therapeutic products into and through
clinical trials. If the initial safety study of our lead therapeutic was halted due to safety
concerns, this would negatively affect the value of our stock.
The results of early Phase 1 trials are based on a small number of patients over a short
period of time, and our progress may not be indicative of results in a large number of patients or
of long-term efficacy. The results in early phases of clinical testing are based upon limited
numbers of patients and a limited follow-up period. For example, the initial results from the Phase
1 clinical trial of our ZFP Therapeutic, SB-509 product, became available in the first half of
2006. The primary end point of the trial was clinical and laboratory safety, however we collected
some preliminary efficacy data that showed trends of clinical improvement in some subjects.
Typically, our Phase 1 clinical trials for indications of safety enroll less than 50 patients. We
anticipate that our Phase 2 clinical trials for safety and efficacy would typically enroll
approximately 100 patients. Actual results with more data points may not confirm favorable results
from earlier stage trials. A number of companies in the pharmaceutical and biotechnology industries
have suffered
18
significant setbacks in late stage clinical trials even after achieving promising
results in earlier stage clinical trials. In addition, we do not yet know if early results will be
reproducible. If a larger population of patients does not experience positive results, or if these
results are reproducible, our products may not receive approval from the FDA. Failure to
demonstrate the safety and effectiveness of our ZFP Therapeutic products in larger patient
populations could have a material adverse effect on our business that would cause our stock price
to decline significantly.
We have limited experience in conducting clinical trials. Our ZFP Therapeutics may fail to
show the desired safety and efficacy in initial clinical trials. We have completed a Phase 1 trial
and begun a Phase 2 clinical trial, however, the FDA will require additional clinical testing which
involves significantly greater resources, commitments and expertise that may require us to enter
into a collaborative relationship with a pharmaceutical company that could assume responsibility
for late-stage development and commercialization.
We may not be able to find acceptable patients or may experience delays in enrolling patients
for our clinical trials. We or the FDA may suspend our clinical trials at any time if either
believes that we are exposing the subjects participating in these trials to unacceptable health
risks. The FDA or institutional review boards and/or institutional biosafety committees at the
medical institutions and healthcare facilities where we sponsor clinical trials may suspend any
trial indefinitely if they find deficiencies in the conduct of these trials. The FDA and
institutional review boards may also require large numbers of patients, and the FDA may require
that we repeat a clinical trial.
Our potential therapeutic products are subject to a lengthy and uncertain regulatory process,
and we may encounter unanticipated toxicity or adverse events or fail to demonstrate efficacy,
causing us to delay, suspend or terminate the development of a ZFP Therapeutics. If these
potential products are not approved, we will not be able to commercialize those products. The FDA
must approve any human therapeutic product before it can be marketed in the United States. The
process for receiving regulatory approval is long and uncertain, and a potential product may not withstand the rigors of testing under the regulatory approval
processes.
Before commencing clinical trials in humans, we must submit an Investigational New Drug (IND)
application to the FDA. The FDA has 30 days to comment on the IND. If the FDA does not comment on
the IND, we or our commercial partner may begin clinical trials.
Clinical trials are subject to oversight by institutional review boards and the FDA. In
addition, our proposed clinical studies will require review from the Recombinant DNA Advisory
Committee, or RAC, which is the advisory board to the National Institutes of Health, or NIH,
focusing on clinical trials involving gene transfer. We will typically submit a proposed clinical
protocol and other product-related information to the RAC three to six months prior to the expected
IND filing date.
Clinical trials:
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must be conducted in conformance with the FDAs good clinical practices ICH guidelines
and other applicable regulations; |
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must meet requirements for institutional review board (IRB) oversight; |
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must follow Institutional Biosafety Committee (IBC) and NIH RAC guidelines where applicable; |
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must meet requirements for informed consent; |
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are subject to continuing FDA oversight; |
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may require large numbers of test subjects; and |
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may be suspended by a commercial partner, the FDA, or us at any time if it is believed
that the subjects participating in these trials are being exposed to unacceptable health
risks or if the FDA finds deficiencies in the IND or the conduct of these trials. |
Clinical trials are lengthy and are typically conducted in three sequential phases, but the
phases may overlap or be combined. Each trial must be reviewed and approved by an independent
ethics committee or institutional review board before it can begin. Phase 1 usually involves the
initial introduction of the investigational drug into healthy volunteers or patients to evaluate
certain factors, including its safety, dosage tolerance and, if possible, to gain an early
indication of its effectiveness. Phase 2 usually involves trials in a limited patient population to
evaluate dosage tolerance and appropriate dosage, identify possible adverse effects and safety
risks, and
19
evaluate preliminarily the efficacy of the drug for specific indications. Phase 3 trials
usually further evaluate clinical efficacy and test further for safety by using the drug in its
final form in an expanded patient population. Later clinical trials may fail to support the
findings of earlier trials, which would delay, limit or prevent regulatory approvals.
While we have stated our intention to file additional IND applications during the next several
years, this is only a statement of intent, and we may not be able to do so because the associated
product candidates may not meet the necessary preclinical requirements. In addition, there can be
no assurance that, once filed, an IND application will result in the actual initiation of clinical
trials.
We cannot predict whether or when we will obtain regulatory approval to commercialize our
product candidates, therefore we cannot predict the timing of any future revenue from these product
candidates. We cannot commercialize any of our ZFP Therapeutics to generate revenue until the
appropriate regulatory authorities have reviewed and approved the applications for the product
candidates. We cannot assure you that the regulatory agencies will complete their review processes
in a timely manner or that we will obtain regulatory approval for any product candidate that we or
our collaborators develop. Satisfaction of regulatory requirements typically takes many years, is
dependent upon the type, complexity and novelty of the product and requires the expenditure of
substantial resources. Regulatory approval processes outside the United States include all of the
risks associated with the FDA approval process. In addition, we may experience delays or rejections
based upon additional government regulation from future legislation or administrative action or
changes in FDA policy during the period of product development, clinical trials and FDA regulatory
review.
Our collaborators may control aspects of our clinical trials, which could result in delays and
other obstacles in the commercialization of our proposed products. For some programs we may be
dependent on third party collaborators to design and conduct our clinical trials. As a result, we
may not be able to conduct these programs in the manner or on the time schedule we currently
contemplate. In addition, if any of these collaborative partners withdraw support for our programs
or proposed products or otherwise impair their development, our business could be negatively
affected.
Our gene regulation and gene modification technology is relatively new, and if we are unable
to use this technology in all our intended applications, it would limit our revenue opportunities.
Our technology involves a relatively new approach to gene regulation and gene modification.
Although we have generated ZFP TFs for thousands of gene sequences, we have not created ZFP TFs for
all gene sequences and may not be able do so, which could limit the usefulness of our technology.
In addition, while we have demonstrated the function of engineered ZFP TFs in mammalian cell
culture, yeast, insects, plants, and animals, we have not yet definitively done so in humans, and
the failure to do so could restrict our ability to develop commercially viable products. If we, and
our collaborators or strategic partners, are unable to extend our results to new commercially
important genes, experimental animal models, and human clinical studies, we may be unable to use
our technology in all its intended applications. Also, delivery of ZFP TFs and ZFNs into cells and
organisms, including humans, in these and other environments is limited by a number of technical
hurdles, which we may be unable to surmount. This is a particular challenge for therapeutic
applications of our technology that will require the use of gene transfer systems that may not be
effective for the delivery of our ZFP TFs or ZFNs in a particular therapeutic application.
The expected value and utility of our ZFP TFs and ZFNs is in part based on our belief that the
targeted or specific regulation of gene expression and targeted gene modification may enable us to
develop a new therapeutic approach as well as to help scientists better understand the role of
human, animal, and other genes in disease and to aid their efforts in drug discovery and
development. We also believe that the regulation of gene expression and targeted gene addition will
have utility in agricultural applications. There is only a limited understanding of the role of
specific genes in all these fields. Life sciences companies have developed or commercialized only a
few products in any of these fields based on results from genomic research or the ability to
regulate gene expression. We, our collaborators, or our strategic partners, may not be able to use
our technology to identify and validate drug targets or to develop commercial products in the
intended markets.
We are currently engaged in the research and development of a new application of our
technology platform: ZFP-mediated gene modification using ZFNs to effect gene disruption, gene correction or gene
addition. Using this technique, Sangamo scientists have engineered ZFNs to cut DNA at a specific
site within a target gene, and to rejoin the two ends of the break which frequently results in the
disruption of the genes function; to correct the adjacent sequences with newly synthesized DNA
copied from an introduced DNA template, resulting in gene correction; or to specifically add a new
DNA sequence into a target site. ZFP-mediated gene modification is at an early stage of
development. Our scientists have shown ZFP-mediated gene modification to work in isolated cells;
however, a significant amount of additional research will be needed before this technique can be
evaluated in animals or plants and subsequently tested for applications in human healthcare and
plant agriculture.
20
We may be unable to license gene transfer technologies that we may need to commercialize our
ZFP TF technology. In order to regulate a gene in a cell, the ZFP TF or ZFN must be efficiently
delivered to the cell. We have licensed certain gene transfer technologies for use with our
Enabling Technologies, which are ZFP TFs and ZFNs used in pharmaceutical discovery research and
protein production. We are evaluating these systems and other technologies that may need to be used
in the delivery of ZFP TFs or ZFNs into cells for in vitro and in vivo applications, including ZFP
Therapeutics. However, we may not be able to license the gene transfer technologies required to
develop and commercialize our ZFP Therapeutics. We have not developed our own gene transfer
technologies, and we rely on our ability to enter into license agreements to provide us with rights
to the necessary gene transfer technology. The inability to obtain a license to use gene transfer
technologies with entities which own such technology on reasonable commercial terms, if at all,
could delay or prevent the preclinical evaluation, clinical testing, and/or commercialization of
our therapeutic product candidates.
We do not currently have the infrastructure or capability to manufacture therapeutic products
on a commercial scale. In order for us to commercialize these products directly, we would need to
develop, or obtain through outsourcing arrangements, the capability to execute all of these
functions. If we are unable to develop or otherwise obtain the requisite preclinical, clinical,
regulatory, manufacturing, marketing, and sales capabilities, we would be unable to directly
commercialize our therapeutics products which would limit our future growth.
Even if our technology proves to be effective, it still may not lead to commercially viable
products. Even if our collaborators or strategic partners are successful in using our ZFP
technology in drug discovery, protein production, therapeutic development, or plant agriculture,
they may not be able to commercialize the resulting products or may decide to use other methods
competitive with our technology. To date, no company has received marketing approval or has
developed or commercialized any therapeutic or agricultural products based on our technology.
Should our technology fail to provide safe, effective, useful, or commercially viable approaches to
the discovery and development of these products, this would significantly limit our business and
future growth and would adversely affect our value.
Even if our product development efforts are successful and even if the requisite regulatory
approvals are obtained, our ZFP Therapeutics may not gain market acceptance among physicians,
patients, healthcare payers and the medical community. A number of additional factors may limit
the market acceptance of products including the following:
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rate of adoption by healthcare practitioners; |
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rate of a products acceptance by the target population; |
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timing of market entry relative to competitive products; |
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availability of alternative therapies; |
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price of our product relative to alternative therapies; |
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availability of third-party reimbursement; |
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extent of marketing efforts by us and third-party distributors or agents retained by us; and |
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side effects or unfavorable publicity concerning our products or similar products. |
Adverse events in the field of gene therapy may negatively impact regulatory approval or
public perception of our potential products. Our potential therapeutic products are delivered to
patients as gene-based drugs, or gene therapy. The clinical and commercial success of our potential
products will depend in part on public acceptance of the use of gene therapy for the prevention or
treatment of human diseases. Public attitudes may be influenced by claims that gene therapy is
unsafe, and, consequently, our products may not gain the acceptance of the public or the medical
community. Negative public reaction to gene therapy in general could result in greater government
regulation and stricter labeling requirements of gene therapy products, including any of our
products, and could cause a decrease in the demand for any products we may develop.
21
Our stock price is also influenced by public perception. Reports of serious adverse events in
a retroviral gene transfer trial for infants with X-linked severe combined immunodeficiency
(X-linked SCID) in France and subsequent FDA actions putting related trials on hold in the United
States had a significant negative impact on the public perception and stock price of certain
companies involved in gene therapy. Stock prices of these companies declined whether or not the
specific company was involved with retroviral gene transfer for the treatment of infants with
X-linked SCID, or whether the specific companys clinical trials were placed on hold in connection
with these events. Other potential adverse events in the field of gene therapy may occur in the
future that could result in greater governmental regulation of our potential products and potential
regulatory delays relating to the testing or approval of our potential products
We are at the development phase of operations and may not succeed or become profitable. We
began operations in 1995 and are in the early phases of ZFP Therapeutic product development. We
have incurred significant losses and our net losses for the past three fiscal years ended 2006,
2005 and 2004 were $17.9 million, $13.3 million and $13.8 million, respectively. To date, our
revenues have been generated from Enabling Technology collaborations, strategic partners, and
federal government research grants. Since 2005, we have placed more emphasis on higher-value
therapeutic product development and related strategic partnerships. This shift in emphasis has the
potential to increase the return on investment to our stockholders by allocating capital resources
to higher value, therapeutic product development activities. At the same time, it increases our
financial risk by increasing expenses associated with product development. In addition, the
preclinical or clinical failure of any single product may have a significant effect on the actual
or perceived value of our shares. Our business is subject to all of the risks inherent in the
development of a new technology, which included the need to:
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attract and retain qualified scientific and technical staff and management, particularly
scientific staff with expertise to develop our early-stage technology into therapeutic products; |
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obtain sufficient capital to support the expense of developing our technology platform
and developing, testing, and commercializing products; |
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develop a market for our products; |
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successfully transition from a company with a research focus to a company capable of
supporting commercial activities; and |
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attract and enter into research collaborations with research and academic institutions
and scientists. |
Commercialization of our technologies will depend, in part, on strategic partnering with other
companies. If we are not able to find strategic partners in the future or our strategic partners do
not diligently pursue product development efforts, we may not be able to develop our technologies
or products, which could slow our growth and decrease our value. We expect to rely, to some
extent, on our strategic partners to provide funding in support of our research and to perform
independent research and preclinical and clinical testing. Our technology is broad based, and we do
not currently possess the resources necessary to fully develop and commercialize potential products
that may result from our technologies or the resources or capabilities to complete the lengthy
marketing approval processes that may be required for the products. Therefore, we plan to rely on
strategic partnerships to help us develop and commercialize ZFP Therapeutic products. If those
partners are unable or unwilling to advance our programs, or if they do not diligently pursue
product approval, this may slow our progress and defer our revenues. Our partners may sublicense or
abandon development programs or we may have disagreements with our partners, which would cause
associated product development to slow or cease. There can be no assurance that we will be able to
establish strategic collaborations for ZFP Therapeutic product development. We may require
significant time to secure collaborations or strategic partners because we need to effectively
market the benefits of our technology to these future collaborators and strategic partners, which
use the time and efforts of research and development personnel and our management. Further, each
collaboration or strategic partnering arrangement will involve the negotiation of terms that may be
unique to each collaborator or strategic partner. These business development efforts may not result
in a collaboration or strategic partnership.
The loss of any future strategic partnering agreements would not only delay or terminate the
potential development or commercialization of products we may derive from our technologies, but it
may also delay or terminate our ability to test ZFP TFs for specific genes. If any strategic
partner fails to conduct the collaborative activities successfully and in a timely manner, the
preclinical or clinical development or commercialization of the affected product candidates or
research programs could be delayed or terminated.
22
Our existing strategic partnering agreements are based on the achievement of milestones. Under
the strategic partnering agreements, we expect to receive revenue for the research and development
of a ZFP Therapeutic product and based on achievement of specific milestones. Achieving these
milestones will depend, in part, on the efforts of our strategic partner as well as our own. In
contrast, our historic Enabling Technology collaborations only pay us to supply ZFP TFs for the
collaborators independent use, rather than for future results of the collaborators efforts. If
we, or any strategic partner, fail to meet specific milestones, then the strategic partnership may
be terminated, which could decrease our revenues.
If our competitors develop, acquire, or market technologies or products that are more
effective than ours, this would reduce or eliminate our commercial opportunity. Any products that
we or our collaborators or strategic partners develop by using our ZFP technology platform will
enter into highly competitive markets. Even if we are able to generate ZFP Therapeutics that are
safe and effective for their intended use, competing technologies may prove to be more effective or
less expensive, which, to the extent these competing technologies achieve market acceptance, will
limit our revenue opportunities. In some cases, competing technologies have proven to be
satisfactorily effective and less expensive, as has been the case with technologies competitive
with our Enabling Technology. The effectiveness of these competing products has reduced the
revenues generated by our Enabling Technology. Competing technologies may include other methods of
regulating gene expression or modifying genes. ZFP TFs and ZFNs have broad application in the life
sciences and compete with a broad array of new technologies and approaches being applied to genetic
research by many companies. Competing proprietary technologies with our product development focus
include:
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small molecule drugs; |
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monoclonal antibodies; |
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recombinant proteins; |
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gene therapy /cDNAs; |
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antisense; and |
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siRNA approaches. |
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For our Enabling Technology Applications: |
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For protein production: gene amplification, meganucleases, insulator technology,
mini-chromosomes; |
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For target validation: antisense, siRNA; and |
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For plant agriculture: recombination approaches, mutagenesis approaches,
meganucleases, mini-chromosomes. |
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In addition to possessing competing technologies, our competitors include biotechnology
companies with: |
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substantially greater capital resources than ours; |
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larger research and development staffs and facilities than ours; and |
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greater experience in product development and in obtaining regulatory approvals and
patent protection. |
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These organizations also compete with us to: |
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attract qualified personnel; |
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attract parties for acquisitions, joint ventures or other collaborations; and |
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license the proprietary technologies of academic and research institutions that are
competitive with our technology, which may preclude us from pursuing similar
opportunities. |
23
Accordingly, our competitors may succeed in obtaining patent protection or commercializing
products before us. In addition, any products that we develop may compete with existing products or
services that are well established in the marketplace.
Our collaborators or strategic partners may decide to adopt alternative technologies or may be
unable to develop commercially viable products with our technology, which would negatively impact
our revenues and our strategy to develop these products. Our collaborators or strategic partners
may adopt alternative technologies, which could decrease the marketability of ZFP technology.
Additionally, because many of our collaborators or strategic partners are likely to be working on
more than one development project, they could choose to shift their resources to projects other
than those they are working on with us. If they do so, this would delay our ability to test our
technology and would delay or terminate the development of potential products based on our ZFP
technology. Further, our collaborators and strategic partners may elect not to develop products
arising out of our collaborative and strategic partnering arrangements or to devote sufficient
resources to the development, manufacturing, marketing, or sale of these products. If any of these
events occur, we may not be able to develop our technologies or commercialize our products.
We anticipate continuing to incur operating losses for the next several years. If material
losses continue for a significant period, we may be unable to continue our operations. We have
incurred operating losses since we began operations in 1995. The extent of our future losses and
the timing of profitability are uncertain, and we expect to incur losses for the foreseeable
future. We have been engaged in developing our ZFP TF technology since inception, which has and
will continue to require significant research and development expenditures. In June 2006, in an
underwritten public offering and pursuant to an effective registration statement, we sold 3,100,000
shares of common stock at a public offering price of $6.75 per share, resulting in net proceeds of
approximately $20.15 million after deducting underwriters discount. In November 2005, we completed
a registered direct offering to institutional and strategic investors for a total of 5,080,000
shares of common stock at a price of $3.85 per share to the investors, resulting in net proceeds to
Sangamo of approximately $18.2 million. To date, we have generated all other revenue from Enabling
Technology collaborations, strategic partnering agreements, federal government research grants and
grants awarded by research foundations. As of March 31, 2007, we had an accumulated deficit of
approximately $133.6 million. We expect to incur losses for the foreseeable future. These losses
will increase as we expand and extend our research and development activities into human
therapeutic product development. If the time required to generate significant product revenues and
achieve profitability is longer than we currently anticipate or if we are unable to generate
liquidity through equity financing, we may not be able to sustain our operations.
We may be unable to raise additional capital, which would harm our ability to develop our
technology and products. We have incurred significant operating losses and negative operating cash
flows since inception and have not achieved profitability. We expect capital outlays and operating
expenditures to increase over the next several years as we expand our infrastructure and research
and ZFP Therapeutic product development activities. While we believe our financial resources will
be adequate to sustain our current operations at least through 2008, we may seek additional sources of capital through equity or debt financing. In addition,
as we focus our efforts on proprietary human therapeutics, we will need to seek FDA approval of
potential products, a process that could cost in excess of $100 million per product. We cannot be
certain that we will be able to obtain financing on terms acceptable to us, or at all. If adequate
funds are not available, our business and our ability to develop our technology and ZFP Therapeutic
products would be harmed.
Our stock price has been volatile and may continue to be volatile, which could result in
substantial losses for investors. During the quarter ended March 31, 2007, our stock price range
from a low of $6.22 to high of $8.85. During the past two years, our common stock price has
fluctuated significantly, ranging from a low of $4.10 to a high of $8.00 during the year ended
December 31, 2006, and a low of $3.46 to a high of $6.49 during the year ended December 31, 2005.
Volatility in our common stock could cause stockholders to incur substantial losses. An active
public market for our common stock may not be sustained, and the market price of our common stock
may continue to be highly volatile. The market price of our common stock has fluctuated
significantly in response to the following factors, some of which are beyond our control:
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announcements by us about the development and
commercialization status of ZFP Therapeutics; |
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changes in market valuations of similar companies; |
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deviations in our results of operations from the guidance given by us or estimates of securities analysts; |
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announcements by us or our competitors of new or enhanced products, technologies or
services or significant contracts, acquisitions, strategic relationships, joint ventures or
capital commitments;
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regulatory developments; |
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additions or departures of key personnel; |
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future sales of our common stock or other securities by the Company, management or
directors; |
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future sale or liquidation of our common stock by
institutional investors with large holding of our stock; and |
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decreases in our cash balances. |
Our common stock is relatively thinly traded, which means large transactions in our common
stock may be difficult to conduct in a short time frame. We have a relatively low volume of daily
trades in our common stock on the Nasdaq Global Market. For example, the average daily trading
volume in our common stock on the Nasdaq Global Market over the ten-day trading period prior to
April 24 , 2007 was approximately 252,690 shares per day. Any large transactions in our common
stock may be difficult to conduct and may cause significant fluctuations in the price of our common
stock.
Failure to attract, retain, and motivate skilled personnel and cultivate key academic
collaborations will delay our product development programs and our research and development
efforts. We are a small company with 80 full-time employees as of April 30, 2007 and our success
depends on our continued ability to attract, retain, and motivate highly qualified management and
scientific personnel and our ability to develop and maintain important relationships with leading
research and academic institutions and scientists. Competition for personnel and academic and other
research collaborations is intense. The success of our technology development programs depends on our ability to attract and retain highly trained personnel. We
have experienced a rate of employee turnover that we believe is typical of emerging biotechnology
companies. If we lose the services of personnel with the necessary skills, it could significantly
impede the achievement of our research and development objectives. We are not presently aware of
any plans of specific employees to retire or otherwise leave the company. If we fail to negotiate
additional acceptable collaborations with academic and other research institutions and scientists,
or if our existing collaborations are unsuccessful, our ZFP Therapeutic development programs may be
delayed or may not succeed.
If conflicts arise between us and our collaborators, strategic partners, scientific advisors,
or directors, these parties may act in their self-interest, which may limit our ability to
implement our strategies. If conflicts arise between our corporate or academic collaborators,
strategic partners, or scientific advisors or directors and us, the other party may act in its
self-interest, which may limit our ability to implement our strategies. Some of our academic
collaborators and strategic partners are conducting multiple product development efforts within
each area that is the subject of the collaboration with us. Our collaborators or strategic
partners, however, may develop, either alone or with others, products in related fields that are
competitive with the products or potential products that are the subject of these collaborations.
Competing products, either developed by the collaborators or strategic partners or to which the
collaborators or strategic partners have rights, may result in the withdrawal of partner support
for our product candidates.
Some of our collaborators or strategic partners could also become competitors in the future.
Our collaborators or strategic partners could develop competing products, preclude us from entering
into collaborations with their competitors, fail to obtain timely regulatory approvals, terminate
their agreements with us prematurely, or fail to devote sufficient resources to the development and
commercialization of products. Any of these developments could harm our product development
efforts.
Because it is difficult and costly to protect our proprietary rights, and third parties have
filed patent applications that are similar to ours, we cannot ensure the proprietary protection of
our technologies and products. Our commercial success will depend in part on obtaining patent
protection of our technology and successfully defending any of our patents that may be challenged.
The patent positions of pharmaceutical and biotechnology companies can be highly uncertain and can
involve complex legal and factual questions. No consistent policy regarding the breadth of claims
allowed in biotechnology patents has emerged to date. Accordingly, we cannot predict the breadth of
claims allowed in patents we own or license.
We are a party to various license agreements that give us rights under specified patents and
patent applications. Our current licenses, as our future licenses frequently will, contain
performance obligations. If we fail to meet those obligations, the licenses could be terminated. If
we are unable to continue to license these technologies on commercially reasonable terms, or at
all, we may be forced to delay or terminate our product development and research activities.
With respect to our present and any future sublicenses, since our rights derive from those
granted to our sublicensor, we are subject to the risk that our sublicensor may fail to perform its
obligations under the master license or fail to inform us of useful improvements in, or additions
to, the underlying intellectual property owned by the original licensor.
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We are unable to exercise the same degree of control over intellectual property that we
license from third parties as we exercise over our internally developed intellectual property. We
do not control the prosecution of certain of the patent applications that we license from third
parties; therefore, the patent applications may not be prosecuted exactly as we desire or in a timely manner.
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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the patents of others will not have an adverse effect on our ability to do business; |
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others will not independently develop similar or alternative technologies or reverse
engineer any of our products, processes or technologies; |
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any of our pending patent applications will result in issued patents; |
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any patents issued or licensed to us or our collaborators or strategic partners will
provide a basis for commercially viable products or will provide us with any competitive
advantages; |
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any patents issued or licensed to us will not be challenged and invalidated by third parties; or |
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we will develop additional products, processes or technologies that are patentable. |
Others have filed and in the future are likely to file patent applications that are similar to
ours. We are aware that there are academic groups and other companies that are attempting to
develop technology that is based on the use of zinc finger and other DNA binding proteins, and that
these groups and companies have filed patent applications. Several patents have been issued,
although we have no current plans to use the associated inventions. If these or other patents
issue, it is possible that the holder of any patent or patents granted on these applications may
bring an infringement action against our collaborators, strategic partners, or us claiming damages
and seeking to enjoin commercial activities relating to the affected products and processes. The
costs of litigating the claim could be substantial. Moreover, we cannot predict whether we, our
collaborators, or strategic partners would prevail in any actions. In addition, if the relevant
patent claims were upheld as valid and enforceable and our products or processes were found to
infringe the patent or patents, we could be prevented from making, using, or selling the relevant
product or process unless we could obtain a license or were able to design around the patent
claims. We can give no assurance that such a license would be available on commercially reasonable
terms, or at all, or that we would be able to successfully design around the relevant patent
claims. There may be significant litigation in the genomics industry regarding patent and other
intellectual property rights, which could subject us to litigation. If we become involved in
litigation, it could consume a substantial portion of our managerial and financial resources.
Third parties have challenged some of our intellectual property and we expect they will continue to
do so. We may not be successful in defending all of our intellectual property that is challenged
which could impede our ability to conduct our business and exclude potential competitors from using
our technology. One of our licensed patents, European Patent No. 0 682 699, entitled Functional
Domains in Flavobacterium Okeanokoites Restriction Endonuclease was granted on May 7, 2003 and
contained claims covering technologies used in our programs in targeted recombination, targeted
integration and gene correction. In December 2005, an interlocutory decision revoking this patent
was issued by the European Patent Office and in March 2007, the European Patent Office upheld its
decision. We do not believe this decision will have a material impact on our ongoing ability, both
in Europe and the United States, to exclude potential competitors in the fields of ZFNs and to
develop, partner and commercialize our ZFP technology.
We rely on trade secrets to protect technology where we believe patent protection is not
appropriate or obtainable. Trade secrets, however, are difficult to protect. While we require
employees, academic collaborators, and consultants to enter into confidentiality agreements, we may
not be able to adequately protect our trade secrets or other proprietary information or enforce
these
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confidentiality agreements.
Our collaborators, strategic partners, and scientific advisors have rights to publish data and
information in which we may have rights. If we cannot maintain the confidentiality of our
technology and other confidential information in connection with our collaborations and strategic
partnerships, then we may not be able to receive patent protection or protect our proprietary
information.
If we do not successfully commercialize certain ZFP Therapeutic programs relating to diabetic
neuropathy under our agreement with JDRF, JDRF may have the right to continue to advance the
program and we may lose control of the intellectual property generated in the collaboration and
development of the product and may only receive a portion of the revenue generated if
commercialization by JDRF is successful. On October 24, 2006, we entered into a Research,
Development and Commercialization Agreement with JDRF. Under the agreement and subject to its
terms and conditions, including our achievement of certain milestones associated with our Phase 2
clinical trial of SB-509 for the treatment of diabetic neuropathy, JDRF will pay us up to
$3,000,000. We are obligated to cover the costs of the Phase 2 trial that are not covered by
JDRFs grant.
Under the agreement, we are obligated to use commercially reasonable efforts to carry out the
Phase 2 trial and, thereafter, to develop and commercialize, a product containing SB-509 for the
treatment of diabetes and complications of diabetes. If we fail to satisfy these obligations, JDRF
may have the right, subject to certain limitations, to obtain an exclusive, sublicensable license,
to the intellectual property generated by us in the course of the Phase 2 trial, to make and
commercialize products containing SB-509 for the treatment of diabetes and complications of
diabetes. If JDRF obtains such a license, it is obligated to pay us a percentage of its revenues
from product sales and sublicensing arrangements. If JDRF fails to satisfy its obligations to
develop and commercialize a product containing SB-509 under the Agreement, then their license
rights will terminate and we will receive a non-exclusive, fully paid license, for any intellectual
property developed during JDRFs use of the license, to research, develop and commercialize
products containing SB-509 for the treatment of diabetes and complications of diabetes. There is
no guarantee that we will be successful in commercializing a product containing SB-509 in the
future. If we fail to do so under the agreement with JDRF, we may lose control of the intellectual
property generated in the development of the product and may only receive a portion of the revenue
generated if commercialization by JDRF is successful.
Regulatory approval, if granted, may be limited to specific uses or geographic areas, which
could limit our ability to generate revenues. Regulatory approval will be limited to the indicated
use for which we can market a product. Further, once regulatory approval for a product is obtained,
the product and its manufacturer are subject to continual review. Discovery of previously unknown problems with a product or
manufacturer may result in restrictions on the product, manufacturer, and manufacturing facility,
including withdrawal of the product from the market. In Japan and Europe, regulatory agencies also
set or approve prices.
Even if regulatory clearance of a product is granted, this clearance is limited to those
specific states and conditions for which the product is useful, as demonstrated through clinical
trials. We cannot ensure that any ZFP Therapeutic product developed by us, alone or with others,
will prove to be safe and effective in clinical trials and will meet all of the applicable
regulatory requirements needed to receive marketing clearance in a given country.
Outside the United States, our ability to market a product is contingent upon receiving a
marketing authorization from the appropriate regulatory authorities, so we cannot predict whether
or when we would be permitted to commercialize our product. These foreign regulatory approval
processes include all of the risks associated with FDA clearance described above.
Our collaborations with outside scientists may be subject to change, which could limit
our access to their expertise. We work with scientific advisors and collaborators at academic
research institutions. These scientists are not our employees and may have other commitments that
would limit their availability to us. Although our scientific advisors generally agree not to do
competing work, if a conflict of interest between their work for us and their work for another
entity arises, we may lose their services. Although our scientific advisors and academic
collaborators sign agreements not to disclose our confidential information, it is possible that
some of our valuable proprietary knowledge may become publicly known through them.
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Laws or public sentiment may limit the production of genetically modified agricultural
products in the future, and these laws could reduce our partners ability to sell these products.
Genetically modified products are currently subject to public debate and heightened regulatory
scrutiny, either of which could prevent or delay production of agricultural products. Effective as
of October 1, 2005, we entered into a Research License and Commercial Option Agreement with DAS.
Under this agreement, we will provide DAS with access to our proprietary ZFP technology and the
exclusive right to use our ZFP technology to modify the genomes or alter the nucleic acid or
protein expression of plant cells, plants, or plant cell cultures. The field-testing, production,
and marketing of genetically modified plants and plant products are subject to federal, state,
local, and foreign governmental regulation. Regulatory agencies administering existing or future
regulations or legislation may not allow production and marketing of our genetically modified
products in a timely manner or under technically or commercially feasible conditions. In addition,
regulatory action or private litigation could result in expenses, delays, or other impediments to
our product development programs or the commercialization of resulting products.
The FDA currently applies the same regulatory standards to foods developed through
genetic engineering as those applied to foods developed through traditional plant breeding.
Genetically engineered food products, however, will be subject to pre-market review if these
products raise safety questions or are deemed to be food additives. Governmental authorities could
also, for social or other purposes, limit the use of genetically modified products created with our
gene regulation technology.
Even if we are able to obtain regulatory approval for genetically modified products, our
success will also depend on public acceptance of the use of genetically modified products including
drugs, plants, and plant products. Claims that genetically modified products are unsafe for
consumption or pose a danger to the environment may influence public attitudes. Our genetically
modified products may not gain public acceptance. The subject of genetically modified organisms has
received negative publicity in the United States and particularly in Europe, and such publicity has
aroused public debate. The adverse publicity in Europe could lead to greater regulation and trade
restrictions on imports of genetically altered products. Similar adverse public reaction in the
United States to genetic research and its resulting products could result in greater domestic
regulation and could decrease the demand for our technology and products.
If we use biological and hazardous materials in a manner that causes injury or violates
laws, we may be liable for damages. Our research and development activities involve the controlled
use of potentially harmful biological materials as well as hazardous materials, chemicals, and
various radioactive compounds typically employed in molecular and cellular biology. We routinely
use cells in culture and gene delivery vectors, and we employ small amounts of radioisotopes in
trace experiments. Although we maintain up-to-date licensing and training programs, we cannot
completely eliminate the risk of accidental contamination or injury from the use, storage,
handling, or disposal of these materials. In the event of contamination or injury, we could be held
liable for damages that result, and any liability could exceed our resources. We currently carry
insurance covering claims arising from our use of these materials. However, if we are unable to
maintain our insurance coverage at a reasonable cost and with adequate coverage, our insurance may
not cover any liability that may arise. We are subject to federal, state, and local laws and
regulations governing the use, storage, handling, and disposal of these materials and specified
waste products. To date, we have not experienced significant costs in complying with regulations
regarding the use of these materials.
Anti-takeover provisions in our certificate of incorporation and Delaware law could make
an acquisition of the Company more difficult and could prevent attempts by our stockholders to
remove or replace current management. Anti-takeover provisions of Delaware law, our certificate of
incorporation and our bylaws may discourage, delay or prevent a change in control of our company, even if a change in
control would be beneficial to our stockholders. 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. In particular, under
our certificate of incorporation our board of directors may issue up to 5,000,000 shares of
preferred stock with rights and privileges that might be senior to our common stock, without the
consent of the holders of the common stock. Moreover, without any further vote or action on the
part of the stockholders, the board of directors would have the authority to determine the price,
rights, preferences, privileges, and restrictions of the preferred stock. This preferred stock, if
it is ever issued, may have preference over, and harm the rights of, the holders of common stock.
Although the issuance of this preferred stock would provide us with flexibility in connection with
possible acquisitions and other corporate purposes, this issuance may make it more difficult for a
third party to acquire a majority of our outstanding voting stock. Similarly, our authorized but
unissued common stock is available for future issuance without stockholder approval.
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In addition, our certificate of incorporation:
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states that stockholders may not act by written consent but only at a
stockholders meeting; |
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establishes advance notice requirements for nominations for election to the board of
directors or proposing matters that can be acted upon at stockholders meetings; and |
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limits who may call a special meeting of stockholders. |
We are also subject to Section 203 of the Delaware General Corporation Law, which provides,
subject to certain exceptions, that if a person acquires 15% of our voting stock, the person is an
interested stockholder and may not engage in business combinations with us for a period of
three years from the time the person acquired 15% or more or our voting stock.
Insiders have substantial control over Sangamo and could delay or prevent a change in
corporate control. The interest of management could conflict with the interest of our other
stockholders. Our executive officers and directors beneficially own, in the aggregate,
approximately 16% of our outstanding common stock. As a result, these stockholders, if they choose
to act together, will be able to have a material impact on all matters requiring stockholder
approval, including the election of directors and approval of significant corporate transactions.
This could have the effect of delaying or preventing a change of control of Sangamo, which in turn
could reduce the market price of our stock.
ITEM 2. UNREGISTERED SALES OF EQUITY SECURITIES AND USE OF PROCEEDS
In June 2006, in an underwritten public offering and pursuant to an effective registration
statement, we sold 3,100,000 shares of common stock at a public offering price of $6.75 per share,
resulting in net proceeds of approximately $20.15 million after deducting underwriters discount.
From the time of receipt through March 31, 2007, Sangamo has used the net proceeds from its
initial public offering, registered direct offering and underwritten public offering of common
stock to invest in short-term and long-term, interest bearing, investment-grade securities and has
used its existing cash balances to fund general operations. The proceeds are being used for general
corporate purposes, including working capital and product development. A portion of the net
proceeds will also be used to acquire or invest in complementary businesses or products or to
obtain the right to use complementary technologies. Sangamo has no agreements or commitments with
respect to any such acquisition and is not currently engaged in any material negotiations with
respect to any such transaction.
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ITEM 6. EXHIBITS
(a) Exhibits:
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31.1
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Rule 13a 14(a)
Certification by President and Chief Executive Officer |
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31.2
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Rule 13a 14(a)
Certification by Principal Financial and Accounting Officer |
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Certification Pursuant to 18 U.S.C. Section 1350. |
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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.
SANGAMO BIOSCIENCES, INC. Dated: May 7, 2007
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/s/ Greg S. Zante
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Greg S. Zante |
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Vice President, Finance and Administration |
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(Principal Financial and Accounting Officer) |
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