Securities and Exchange Commission
Washington, D.C. 20549
FORM 10-Q
(Mark One)
[X] QUARTERLY REPORT UNDER SECTION 13 OR 15(D) OF THE SECURITIES
EXCHANGE ACT OF 1934
For the quarterly period ended September 30, 1999
[ ] 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: 000-26727
BIOMARIN PHARMACEUTICAL INC.
(Exact name of registrant issuer as specified in its charter)
Delaware 68-0397820
(State or other jurisdiction of (I.R.S. Employer Identification No.)
incorporation or organization)
371 Bel Marin Keys Blvd., Suite 210, Novato, California 94949
(address of principal executive offices)
(Zip Code)
(415) 884-6700
(Registrant's telephone number, including area code)
(Former name, former address and former fiscal year,
if changed since last report)
Indicate by check mark whether the registrant (1) has filed all reports
required to be filed by Section 13 or 15(d) of the Securities Exchange Act of
1934 during the past 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 X No
APPLICABLE ONLY TO ISSUERS INVOLVED IN BANKRUPTCY
PROCEEDINGS DURING THE PRECEDING FIVE YEARS
Indicate by check mark whether the registrant filed all documents and
reports required to be filed by Sections 12, 13 or 15(d) of the Securities
Exchange Act of 1934 subsequent to the distribution of securities under a plan
confirmed by a court. Yes ____ No_____
APPLICABLE ONLY TO CORPORATE ISSUERS
Indicate the number of shares outstanding of each of the issuer's classes
of common stock, as of the latest practicable date: 34,821,430 shares Common
Stock, par value $0.001, outstanding as of October 31, 1999.
BIOMARIN PHARMACEUTICAL INC.
TABLE OF CONTENTS
Page
PART I. FINANCIAL INFORMATION
Item 1. Financial Statements (Unaudited).
Consolidated Balance Sheets as of December 31, 1998
and September 30, 1999 2
Consolidated Statements of Operations for the three-month periods
ended September 30, 1998 and 1999 3
Consolidated Statements of Operations for the nine-month periods
ended September 30, 1998 and 1999 and for the period from
March 21, 1997(inception) to September 30, 1999 4
Consolidated Statements of Cash Flows for the nine-month periods
ended September 30, 1998 and 1999 and for the period from
March 21, 1997(inception) to September 30, 1999 5
Notes to Consolidated Financial Statements 6
Item 2. Management's Discussion and Analysis 8
Item 3. Quantitative and Qualitative Disclosure
about Market Risk 25
PART II. OTHER INFORMATION
Item 1. Legal Proceedings 26
Item 2. Changes in Securities and Uses of Proceeds 26
Item 3. Defaults upon Senior Securities 26
Item 4. Submission of Matters to a Vote of Security Holders 26
Item 5. Other Information 26
Item 6. Exhibits and Reports on Form 8-K 27
SIGNATURE 28
PART 1. FINANCIAL INFORMATION
Item 1. Financial Statements (Unaudited)
BIOMARIN PHARMACEUTICAL INC. AND SUBSIDIARIES
(a development-stage company)
CONSOLIDATED BALANCE SHEETS
AS OF DECEMBER 31, 1998 AND SEPTEMBER 30, 1999
($ Thousands, except for share and per share data)
December 31, September 30,
1998 1999
---------------- ---------------
ASSETS (unaudited)
CURRENT ASSETS:
Cash and cash equivalents $ 9,414 $ 32,585
Short-term investments 1,975 39,572
Accounts receivable, net 148 409
Due from Glyko Biomedical Ltd. 114 137
Due from BioMarin/Genzyme LLC 419 2,185
Inventories 72 939
Prepaid expenses 676 529
---------------- ---------------
Total current assets 12,818 76,356
PROPERTY AND EQUIPMENT, net 6,223 22,934
GOODWILL AND OTHER INTANGIBLE ASSETS, net 11,704 11,613
INVESTMENT IN BIOMARIN/GENZYME LLC 685 1,510
DEPOSITS 79 120
---------------- ---------------
Total assets $ 31,509 $ 112,533
================ ===============
LIABILITIES AND STOCKHOLDERS' EQUITY
CURRENT LIABILITIES:
Accounts payable $ 1,340 $ 4,961
Accrued liabilities 640 623
Notes payable short-term 24 26
---------------- ---------------
Total current liabilities 2,004 5,610
LONG-TERM LIABILITIES:
Long-term portion of notes 110 90
---------------- ---------------
Total liabilities 2,114 5,700
STOCKHOLDERS' EQUITY:
Common stock, $0.001 par value:
50,000,000 shares authorized,
26,176,180 and 34,810,129 shares issued
and outstanding at December 31, 1998, and
September 30, 1999, respectively 26 35
Additional paid-in capital 50,058 146,302
Warrants 128 128
Deferred compensation (3,253) (2,922)
Notes receivable from stockholders (2,488) (2,600)
Deficit accumulated during the
development stage (15,076) (34,110)
---------------- ---------------
Total stockholders' equity 29,395 106,833
---------------- ---------------
Total liabilities and
stockholders' equity $ 31,509 $ 112,533
================ ===============
The accompanying notes are an integral part of these statements.
2
BIOMARIN PHARMACEUTICAL INC. AND SUBSIDIARIES
(a development-stage company)
CONSOLIDATED STATEMENTS OF OPERATIONS
FOR THE THREE-MONTH PERIODS ENDED SEPTEMBER 30, 1998 AND 1999
($ Thousands, except for per share data)
Three Months Ended
September 30,
------------------------------------
1998 1999
--------------- --------------
(unaudited) (unaudited)
REVENUES:
Revenues--products $ -- $ 489
Revenues--services -- 3
Revenues from BioMarin/Genzyme LLC 141 1,507
Revenues--other --
--------------- --------------
Total revenues 141 1,999
OPERATING COSTS AND EXPENSES:
Cost of products -- 117
Cost of services -- 1
Research and development 1,749 7,658
Selling, general and administrative 1,440 1,955
--------------- --------------
Total operating costs
and expenses 3,189 9,731
--------------- --------------
Loss from operations (3,048) (7,732)
INTEREST INCOME 230 724
INTEREST EXPENSE -- (167)
EQUITY IN LOSS OF BIOMARIN/GENZYME LLC -- (468)
--------------- --------------
Net loss $ (2,818) $ (7,643)
=============== ==============
NET LOSS PER SHARE, basic and diluted $ (0.12) $ (0.24)
=============== ==============
WEIGHTED AVERAGE COMMON SHARES
OUTSTANDING (Thousands) 22,736 32,476
=============== ==============
The accompanying notes are an integral part of these statements.
3
BIOMARIN PHARMACEUTICAL INC. AND SUBSIDIARIES
(a development-stage company)
CONSOLIDATED STATEMENTS OF OPERATIONS
FOR THE NINE-MONTH PERIODS ENDED SEPTEMBER 30, 1998 AND 1999, AND
FOR THE PERIOD FROM MARCH 21, 1997 (INCEPTION) TO SEPTEMBER 30,
1999 ($ Thousands, except for per share data)
For the period
March 31, 1997
Nine Months Ended (Inception), to
September 30, September 30,
---------------------------------------
1998 1999 1999
----------------- ------------------ ----------------
(unaudited) (unaudited) (unaudited)
REVENUES:
Revenues--products $ -- $ 1,018 $ 1,156
Revenues--services -- 81 193
Revenues from BioMarin/Genzyme LLC 141 3,411 4,248
Revenues--other -- 151 254
----------------- ------------------ ----------------
Total revenues 141 4,661 5,851
OPERATING COSTS AND EXPENSES:
Cost of products -- 234 283
Cost of services -- 99 158
Research and development 3,904 18,029 30,446
Selling, general and administrative 2,772 4,759 9,203
----------------- ------------------ ----------------
Total operating costs and
expenses 6,676 23,121 40,090
----------------- ------------------ ----------------
Loss from operations (6,535) (18,460) (34,239)
INTEREST INCOME 469 1,177 1,926
INTEREST EXPENSE -- (728) (728)
EQUITY IN LOSS OF BIOMARIN/GENZYME LLC -- (1,023) (1,070)
----------------- ------------------ ----------------
Net loss $ (6,066) $ (19,034) $ (34,111)
================= ================== ================
NET LOSS PER SHARE, basic and diluted $ (0.28) $ (0.67) $ (1.72)
================= ================== ================
WEIGHTED AVERAGE COMMON SHARES OUTSTANDING
(Thousands) 21,298 28,299 19,801
================= ================== ================
The accompanying notes are an integral part of these statements.
4
BIOMARIN PHARMACEUTICAL INC. AND SUBSIDIARIES
(a development-stage company)
CONSOLIDATED STATEMENTS OF CASH FLOWS
FOR THE NINE-MONTH PERIODS ENDED SEPTEMBER 30, 1998 AND 1999, AND FOR
THE PERIOD FROM MARCH 21, 1997 (INCEPTION) TO SEPTEMBER 30, 1999
($ Thousands)
Period from
Nine Months Ended March 21, 1997
September 30, (Inception), to
1998 1999 September 30, 1999
----------------- ---------------- -----------------
(unaudited) (unaudited) (unaudited)
CASH FLOWS FROM OPERATING ACTIVITIES:
Net loss $ (6,066) $ (19,034) $ (34,111)
Adjustments to reconcile net loss to net cash used in
operating activities:
Depreciation 98 2,353 2,666
Amortization of deferred compensation 58 965 1,228
Amortization of goodwill -- 841 1,112
Accrued interest on notes receivable from stockholders (171) (112) (377)
Compensation in the form of common stock and common
stock options -- -- 18
Loss from BioMarin/Genzyme LLC 142 4,433 4,480
Write-off of in-process technology -- -- 2,625
Changes in operating assets and liabilities:
Accounts receivable (37) (261) (409)
Due from Glyko Biomedical Ltd. (2) (23) (137)
Due from BioMarin/Genzyme LLC (283) (1,766) (2,185)
Inventories -- (867) (939)
Prepaid expenses (79) 148 (528)
Deposits (62) (41) (120)
Accounts payable 1,372 3,620 4,960
Accrued liabilities (43) (17) 623
Due to Glyko, Inc. 94 -- --
--------------- ---------------- -----------------
Net cash used in operating activities (4,979) (9,761) (21,094)
--------------- ---------------- -----------------
CASH FLOWS FROM INVESTING ACTIVITIES:
Purchase of property and equipment (1,897) (19,063) (25,597)
Investment in BioMarin/Genzyme LLC (489) (5,260) (5,992)
Purchase of assets from OGS -- (1,500) (1,500)
Intangibles and other assets, net -- 750 750
Sale (purchase) of short-term investments 901 (37,596) (39,572)
--------------- ---------------- -----------------
Net cash used in investing activities (1,485) (62,669) (71,911)
--------------- ---------------- -----------------
CASH FLOWS FROM FINANCING ACTIVITIES:
Proceeds from note payable -- -- 134
Bridge loan -- -- 880
Proceeds from sale of common stock, net of issuance costs 19,430 95,619 124,594
Repayment of equipment loan -- (18) (18)
--------------- ---------------- -----------------
Net cash provided by financing activities 19,430 95,601 125,590
--------------- ---------------- -----------------
Net increase in cash and cash equivalents 12,966 23,171 32,585
CASH AND CASH EQUIVALENTS:
Beginning of period 5,987 9,414 --
--------------- ---------------- -----------------
End of period $ 18,953 $ 32,585 $ 32,585
=============== ================ =================
The accompanying notes are an integral part of these statements.
5
BIOMARIN PHARMACEUTICAL INC. AND SUBSIDIARIES
(a development-stage company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
(Unaudited)
1. BASIS OF PRESENTATION:
BioMarin Pharmaceutical Inc. (BioMarin or Company) is a developer of
carbohydrate enzyme therapies for debilitating, life-threatening, chronic
genetic disorders and other diseases or conditions. The Company was incorporated
in October 1996 as a wholly-owned subsidiary of Glyko Biomedical Ltd. (GBL). The
Company was funded by GBL and began operations on March 21, 1997, the date of
inception. In October 1998, the Company acquired Glyko, Inc., a wholly-owned
subsidiary of GBL in a transaction valued at $14.5 million.
The accompanying unaudited consolidated financial statements have been prepared
in accordance with generally accepted accounting principles for interim
financial information on substantially the same basis as the annual audited
financial statements. Accordingly, they do not include all of the information
and footnotes required by generally accepted accounting principles for complete
financial statements. In the opinion of management, all adjustments, consisting
of normal recurring adjustments, considered necessary for a fair presentation
have been included. Operating results for the three- and nine-month periods
ended September 30, 1999 are not necessarily indicative of the results that may
be expected for the year ending December 31, 1999. These consolidated financial
statements should be read in conjunction with the financial statements and
footnotes thereto for the year ended December 31, 1998 included in the Company's
Form S-1 Registration Statement.
2. SIGNIFICANT ACCOUNTING POLICIES:
Short-term investments
Short-term investments consists primarily of government and fixed income
securities for which the company plans to hold to maturity. These investments
are reported at amortized costs in the accompanying balance sheets. The total
market value of these securities as of September 30, 1999 was not materially
different from the carrying value.
Property and Equipment
Property and equipment are stated at cost. Depreciation is computed using the
straight-line method. Leasehold improvements are amortized over the life of the
asset or the term of the lease, whichever is shorter. Significant additions and
improvements are capitalized, while repairs and maintenance are charged to
expense as incurred.
Property and equipment consisted of the following ($thousands):
December 31, September 30, Estimated
1998 1999 Useful Lives
------------------ ------------------ -------------------------
Computer hardware and software $ 162 $ 404 3 years
Office furniture and equipment 372 750 5 years
Manufacturing/Laboratory equipment 3,469 7,180 5 years
Shorter of life of
Leasehold improvements 2,532 17,268 asset or lease term
------------------ ------------------
6,535 25,602
Less: Accumulated depreciation (312) (2,668)
------------------ ------------------
Total, net $ 6,223 $ 22,934
================== ==================
6
Research and Development
Research and development expenses include the expenses associated with contract
research and development provided by third parties, research and development
provided in connection with BioMarin/Genzyme LLC, a joint venture, including
clinical and regulatory costs, and internal research and development costs. All
research and development costs discussed above are expensed as incurred.
Net Income (Loss) per Share
Basic net income (loss) per share is calculated by dividing net income (loss) by
the weighted average common shares outstanding during the period. Diluted net
income per share is calculated by dividing net income by the weighted average of
common stock outstanding and potential common shares during the period.
Potential common shares include dilutive shares issuable upon the exercise of
outstanding common stock options, warrants, and contingent issuances of common
stock. For periods in which the Company has losses, such potential common shares
are excluded from the computation of diluted net loss per share, as their effect
is antidilutive.
3. INITIAL PUBLIC OFFERING:
The Company completed its Initial Public Offering (IPO) of 4,500,000 shares of
common stock at $13.00 per share on July 23, 1999, raising net proceeds of
approximately $51.9 million. In a private placement concurrent with the IPO,
Genzyme invested in the Company $10 million at the IPO price of $13 per share
(769,230 shares of common stock). In addition, the $26 million of convertible
notes sold by the Company on April 13, 1999, plus accrued interest, were
converted into 2,672,020 shares of common stock at $10 per share. The
underwriters' over-allotment exercise in August raised additional net proceeds
of $8.1 million at the IPO price (675,000 shares of common stock).
7
Item 2. Management's Discussion and Analysis of Financial
Condition and Results of Operations
FORWARD-LOOKING STATEMENTS
The following discussion and analysis of financial condition and results of
operations contains "forward-looking statements" as defined under securities
laws. These statements can often be identified by the use of terminology such as
"believes," "expects," "anticipates," "plans," "may," "will," "projects,"
"continues," "estimates," "potential," "opportunity" and so on. These
forward-looking statements may be found in the "Risk Factors," and other
sections of this document. Our actual results or experience could differ
significantly from the forward-looking statements. Factors that could cause or
contribute to these differences include those discussed in "Risk Factors," as
well as those discussed elsewhere in this document.
Overview
We are a developer of carbohydrate enzyme therapies for debilitating,
life-threatening, chronic genetic disorders and other diseases or conditions.
Since our inception on March 21, 1997, we have been engaged in research and
development activities, including preclinical studies, clinical trials and
clinical manufacturing, the establishment of laboratory and manufacturing
facilities, and administrative activities. BioMarin was incorporated in October
1996 as a wholly-owned subsidiary of Glyko Biomedical Ltd. ("GBL", TSE: GBL).
BioMarin was initially funded by GBL and began operations on March 21, 1997, the
date of inception.
We have incurred net losses since inception and had an accumulated deficit
through September 30, 1999 of $34.1 million. Our losses have resulted primarily
from research and development activities and related administrative expenses. We
expect to continue to incur operating losses at least six months into 2001.
To date, we have not generated revenues from the sale of our drug candidates.
Our lead product is Aldurazyme(TM), alronidase for injection, (recombinant human
(alpha)-L-iduronidase), which is under clinical trials for use in enzyme
replacement therapy for Mucopolysaccharridosis-I or MPS-I. In previous
documents, Aldurazyme(TM) was identified as BM101. Our financial results may
vary depending on many factors, including:
. The progress of Aldurazyme(TM) in the regulatory
processes and initial sales activities
. The investment in manufacturing process
development and in manufacturing capacity for
Aldurazyme(TM) and other product candidates
. The acceleration of our other pharmaceutical
candidates into preclinical studies and clinical trials
. The progress of our additional research and
development efforts
In September 1998, we established a joint venture with Genzyme for the worldwide
development and commercialization of Aldurazyme(TM) for the treatment of MPS-I.
Under the agreement, our company and Genzyme are each required to make capital
contributions to the joint venture equal to 50% of the expenses associated with
the development and commercialization of Aldurazyme(TM). We will share equally
in any profits generated from the sales of Aldurazyme(TM).
In October 1998, we acquired Glyko, Inc., a wholly-owned subsidiary of GBL in a
transaction valued at $14.5 million. Glyko, Inc. provides products and services
that perform carbohydrate analysis and medical diagnosis to research
institutions and commercial laboratories.
In July 1999, we completed our initial public offering ("IPO") of 4,500,000
shares of our common stock at $13 per share raising net proceeds of
approximately $51.9 million. In a private placement concurrent with the IPO,
Genzyme purchased $10 million of our common stock (769,230 shares) at the IPO
price of $13. In addition, the $26 million of convertible notes sold by the
Company on April 13, 1999, plus accrued interest, were converted into 2,672,020
shares of common stock at $10 per share.
In August 1999, the underwriters exercised their over-allotment option for
675,000 shares at the IPO price of $13 per share, raising additional net
proceeds of $8.1 million.
8
Results of Operations
The Quarters Ended September 30, 1999 and 1998
Revenues for the third quarter of 1999 totaled $1,999,000 compared to revenues
of $141,000 in the third quarter of 1998. Third quarter 1999 revenues included
$1,507,000 from the joint venture with Genzyme General (Nasdaq: GENZ) for the
development and commercialization of Aldurazyme(TM), alronidase for injection,
(recombinant human (alpha)-L-iduronidase) for the treatment of
Mucopolysaccharidosis-I (MPS-I). MPS-I is a chronic, debilitating genetic
disease which afflicts children and leads to death before adulthood in a
majority of patients. The BioMarin/Genzyme joint venture was formed on September
4, 1998. Third quarter 1999 revenues also included $492,000 generated by Glyko,
Inc., BioMarin's subsidiary engaged in the sale of analytical and diagnostic
products and services, which was acquired by BioMarin on October 7, 1998. On an
ongoing basis, Glyko, Inc. revenues for the third quarter of 1999 were up 58% in
comparison to the third quarter of 1998 as a result of revenues from the
acquisition of the biochemical reagents business of Oxford GlycoSciences Plc.
(LSE: OGS), which was acquired in May 1999.
Cost of products and cost of services related to Glyko, Inc. operations were
$118,000 in the third quarter of 1999 and were zero in the comparable period of
1998. On an ongoing basis, Glyko's total product and service costs as a percent
of the sales of products and services were 25% in both the third quarter of 1999
and of 1998.
Research and development expenses for the third quarter of 1999 increased by
$5,909,000 from $1,749,000 in the third quarter of 1998 to $7,658,000 in the
third quarter of 1999. This increase was due primarily to increased activity in
support of the joint venture with Genzyme for Aldurazyme(TM) and in support of
the Company's enzyme product candidates for MPS-VI and burn debridement.
Selling, general and administrative expenses increased from $1,440,000 in the
third quarter of 1998 to $1,955,000 in the third quarter of 1999 due to
increased BioMarin administrative staff expenses to support expanded operations.
BioMarin's equity in the loss of its joint venture with Genzyme was $468,000 for
the third quarter 1999 while BioMarin did not have a loss from the joint venture
in the third quarter of 1998.
Interest income increased by $494,000 from $230,000 in the third quarter of 1998
to $724,000 in the third quarter of 1999 due to increased cash reserves
resulting from the IPO on July 23, 1999, a concurrent investment by Genzyme, and
a convertible note financing in April 1999 that converted at the time of the
initial public offering. The interest expense on the convertible notes, accrued
at an interest rate of 10% per year, totaled $167,000 for the third quarter of
1999.
The net loss was $2,818,000 ($0.12 per share) in the third quarter of 1998 and
increased to $7,643,000 ($0.24 per share) in the comparable period of 1999.
9
The Nine Months Ended September 30, 1999 and 1998
For the nine-month periods ended September 30, 1998 and 1999, revenues were
$141,000 and $4,661,000, respectively. The reasons for this increase in revenues
were the same as described for the third quarter increase in revenues. Joint
venture revenues were $3,411,000 and Glyko, Inc. revenues were $1,218,000 for
the first nine months of 1999.
Research and development expenses increased from $3,904,000 in the first nine
months of 1998 to $18,029,000 in the comparable period of 1999. Increased
expenses in support of the Aldurazyme joint venture with Genzyme, the MPS-VI
program and the burn debridement program were the major factors in the growth of
research and development expenses.
Selling, general and administrative expenses increased from $2,772,000 in the
nine months of 1998 to $4,759,000 in the first nine months of 1999. This
increase resulted from the consolidation of Glyko, Inc. selling and
administrative expenses in 1999 expenses, an increase in staffing in BioMarin
administration in 1999 compared to 1998, and an increase in facilities expense
charged to administration in 1999. The increase in administrative staff and
related expense was necessary to support expanded operations.
BioMarin's equity in the loss of its joint venture with Genzyme was $1,023,000
for the first nine months of 1999 while there was no loss in the comparable
period of 1998.
Interest income increased by $708,000 from $469,000 in the first nine months of
1998 to $1,177,000 in the first nine months of 1999 primarily due to increased
cash reserves resulting from a convertible note financing in April 1999, the
initial public offering, and a concurrent private placement with Genzyme in July
1999. The interest expense accrued on the convertible notes prior to their
conversion in the initial public offering totaled $720,000.
The net loss was $6,066,000 ($0.28 per share) and $19,034,000 ($0.67 per share)
for the first nine months of 1998 and 1999, respectively.
Liquidity and Capital Resources
We have financed our operations since our inception by the issuance of common
stock and convertible notes and the related interest income earned on cash
balances available for short-term investment. Since inception, we have raised
aggregate estimated net proceeds of $125.5 million after the IPO and concurrent
Genzyme investment. We were initially funded by GBL with a $1.5 million
investment. We have since raised additional capital from the sale of common
stock in private placements, the sale of promissory notes convertible into
common stock, an investment of $8.0 million by Genzyme as part of our joint
venture with them, an initial public offering including the underwriters'
over-allotment exercise and the concurrent $10 million Genzyme investment in our
Company.
Our combined cash, cash equivalents and short-term investments totaled $72.2
million at September 30, 1999, an increase of $60.8 million from December 31,
1998. The primary source of cash was the issuance of common stock at our IPO,
generating $60.0 million of net proceeds including the underwriters
over-allotment. The primary use of cash during the nine months ended September
30, 1999 was to finance operations and to purchase leasehold improvements and
equipment. For the nine months ended September 30, 1999, operations used $9.8
million, we purchased $19.1 million of leasehold improvements and equipment,
invested $5.3 million in the joint venture (which was consumed in joint venture
operations) and purchased $1.5 million of assets from Oxford GlycoSciences.
From our inception through September 30, 1999, we have purchased approximately
$25.6 million of leasehold improvements and equipment. We expect that our
investment in leasehold improvements and equipment will increase significantly
during the next two years because we will provide facilities and equipment for a
larger staff and increase manufacturing capacity.
As part of the acquisition of Glyko, Inc., we acquired in-process research and
development projects, the value of which was expensed as a portion of the
purchase price at the time of the acquisition. The 11 projects acquired are each
relatively small and can be grouped into two categories, analytic projects and
diagnostic projects.
10
The analytic projects are intended to expand the analytic product line by adding
new enzymes for reagent sales, new kits for agricultural applications, new
instrument capabilities for protein analysis and a major upgrade of software
capabilities. At the time of the acquisition of Glyko, Inc., all of the analytic
projects had completed feasibility work and the software projects were 75%
complete and have since been completed. The development of specialized materials
supporting instrument capabilities is deemed to be the most difficult technical
hurdle for the completion and commercialization of the analytic projects. The
fair value of the analytic projects was $1.7 million at the time of the
acquisition.
The diagnostic projects are intended to expand a product line based on very
precise measurements of the level of complex carbohydrates in blood and urine as
indicators of serious disease conditions including heart disease, kidney disease
and mucopolysaccharidoses or carbohydrate storage diseases. At the time of the
Glyko, Inc. acquisition, preliminary feasibility work had been done for all of
the projects and a software project was well advanced as to programming, which
has since been completed. The development of new more sensitive carbohydrate
chemistry techniques is deemed to be the most difficult technical hurdle for the
completion and commercialization of the diagnostic products. The fair value of
the diagnostic projects was $924,000 at the time of the acquisition.
As of September 30, 1999, we had expended to date approximately $695,000 on the
in-process research and development projects and $725,000 on the diagnostic
projects. If all acquired in-process research and development projects proceed
to completion, we expect to spend approximately $415,000 in incremental direct
expense to complete the analytic projects in phases over approximately 18
months. We expect to spend approximately $1.0 million to complete the diagnostic
projects in phases completed from 6 to 18 months in the future. None of these
projects have been terminated to date.
Since the acquisition of these in-process research and development projects one
year ago, there have been no subsequent developments which indicate that the
completion and commercialization of either of the projects are less likely to be
completed on the original planned schedule or less likely to be a commercial
success.
We have made and plan to make substantial commitments to capital projects,
including the Aldurazyme(TM) manufacturing facility in Torrance, California, a
manufacturing facility in Novato, and new research and development facilities in
Novato.
In September 1998, we established a joint venture with Genzyme for the worldwide
development and commercialization of Aldurazyme(TM) for the treatment of MPS-I.
We will share expenses and profits from the joint venture equally with Genzyme.
Genzyme purchased $8.0 million in common stock upon signing the agreement and
$10.0 million of common stock at the IPO price of $13 per share in a private
placement concurrent with the IPO. Genzyme has committed to pay us an additional
$12.1 million upon approval of the biologics license application for
Aldurazyme(TM).
On October 7, 1998, we purchased Glyko, Inc. from GBL for an aggregate purchase
price of $14.5 million. The purchase price was paid by 2,259,039 shares of our
common stock, our assumption of certain stock options held by Glyko, Inc.
employees which were exercisable into a maximum of 255,540 shares of our common
stock and $500 in cash.
On April 13, 1999, we sold a total of $26.0 million of convertible promissory
notes. The notes were converted into 2,672,020 shares of our common stock at a
conversion price of $10.00 per share concurrent with the IPO.
In May 1999, Glyko, Inc. acquired key assets of the Biochemical Research Reagent
Division of Oxford GlycoSciences Plc. The acquisition was made to increase
Glyko, Inc.'s product offerings and was valued from $1.5 million to $2.1
million, depending on the future sales of the acquired products.
In July 1999, we completed our initial public offering of 4,500,000 shares of
our common stock at $13 per share raising net proceeds of approximately $51.9
million. In a private placement concurrent with the IPO, Genzyme purchased $10
million of our common stock (769,230 shares) at the IPO price of $13.
11
In August 1999, the underwriters exercised their over-allotment option for
675,000 shares at the IPO price of $13 raising additional net proceeds of $8.1
million.
We expect our current funds to last for a period of at least12 months. Until we
can generate sufficient levels of cash from our operations, we expect to
continue to finance future cash needs through:
. The sale of equity securities
. Equipment-based financing
. Collaborative agreements with corporate partners
We do not expect to generate positive internal cash flow for at least the next
two years because we expect to increase operational expenses and manufacturing
investment for the joint venture and to increase research and development
activities, including:
. Preclinical studies, clinical trials and regulatory review
. Commercialization of our drug candidates
. Development of manufacturing operations
. Process development
. Scale-up of manufacturing facilities
. Sales and marketing activities
We anticipate a need for additional financing to fund the future operations of
its business, including the commercialization of our drug candidates currently
under development. We cannot assure you that additional financing will be
obtained or, if obtained, will be available on reasonable terms.
Our future capital requirements will depend on many factors, including, but not
limited to:
. The progress of our research and development programs
. The progress of preclinical studies and clinical trials
. The time and cost involved in obtaining regulatory approvals
. Scaling up, installing and validating manufacturing capacity
. Competing technological and market developments
. Changes and developments in collaborative, licensing and other
relationships
. The development of commercialization activities and arrangements
. The leasing and build-out of additional facilities
. The purchase of additional capital equipment
We plan to continue our policy of investing available funds in government
securities and investment grade, interest-bearing securities, primarily with
maturities of one year or less. We do not invest in derivative financial
instruments, as defined by Statement of Financial Accounting Standards No. 119.
12
Impact of Year 2000
The following constitutes "Year 2000 Readiness Disclosure" under the Year 2000
Information and Readiness Disclosure Act of 1998.
We are aware of the potential problems associated with computer programs and
systems that use only two digits to identify the year in the date field.
Application and system programs may be unable to correctly process date
information for dates after December 31, 1999. This year 2000 defect could cause
the disruption or failure of computer systems. The year 2000 defect could affect
both our internal information technology systems and other functional systems
that use embedded computer programs for control or other purposes. The defect
could also affect the information technology and other functional systems of
suppliers of products and services to us. The defect could affect the overall
economy and have a significant impact on us.
We have formed a team to review and resolve those aspects of the year 2000
problem which are within our direct control and adjust to or influence those
aspects which are not within our direct control. The team has reviewed our
software products (including those under development) and determined that our
software products do not use date data and are year 2000 compliant. Our
biopharmaceutical products do not have any year 2000 exposure. The team has
reviewed the year 2000 compliance status of our major internal information
technology programs and systems used for administrative requirements and
determined that these systems are year 2000 compliant. We have reviewed the
computer systems used to control our analytical instruments and production
equipment. Due to the recent design of our equipment, the embedded computers are
year 2000 compliant. We believe that the expense of repairing or replacing any
undetected year 2000 defects will not be material. We believe that we can
resolve our internal year 2000 compliance issues before the year 2000 with
expenditures that are currently estimated not to be material. If we do not
achieve on a timely basis year 2000 compliance for our internal systems, our
operations and business could be adversely affected.
With respect to our suppliers, we do not currently process orders, payments and
other business communications electronically from computer to computer. However,
if our suppliers' and ultimate customers' own systems are not yet year 2000
compliant, their disruptions could have a significant direct or indirect impact
on our operations and business. The following consequences of the year 2000
problem could disrupt our business:
. Financial institutions may not be able to process checks,accept deposits,
provide records, process wire transfers, provide stock
ownership and transfer records, or facilitate many other
financial transactions and services.
. Suppliers may not be able to process orders, manufacture products,
deliver in accordance with production schedules, or in general
provide the current level of timely products and services.
. Voice and data communication systems used by us and our customers and
suppliers might be disrupted.
. Health care suppliers and third-party payors may be unable to process
patient records, add to or modify the content of their pharmacy
authorizations, accept or make payments, and handle the many other
data requirements of the modern health care system. The added costs
for back-up systems, for temporary or emergency fixes and the ongoing
requirements to handle critical functions on a timely basis
may delay our introduction of new drugs and therapeutic practices.
13
RISK FACTORS
If we continue to incur operating losses for a period longer than anticipated,
we may be unable to continue our operations.
We are in an early stage of development and have operated at a net loss since we
were formed. Since we began operations in March 1997, we have been engaged
primarily in research and development. We have no sales revenues from any of our
drug products. As of September 30, 1999, we had an accumulated deficit of
approximately $34.1 million. We expect to continue to operate at a net loss at
least six months into 2001. Our future profitability depends on our receiving
regulatory approval of our drug candidates and our ability to successfully
manufacture and market any approved drugs, either by ourselves or jointly with
others. The extent of our future losses and the timing of profitability are
highly uncertain. If we fail to become profitable or are unable to sustain
profitability on a quarterly or annual basis, then we may be unable to continue
our operations.
Because of the relative small size and scale of our wholly-owned subsidiary,
Glyko, Inc., profits from products and services offered by it will be
insufficient to offset the expenses associated with our pharmaceutical business.
As a result, we expect that operating losses will continue and increase for the
foreseeable future.
If we fail to obtain the capital necessary to fund our operations we will be
unable to complete our product development programs.
In the future, we may need to raise substantial additional capital to fund
operations. We cannot be certain that any financing will be available when
needed. If we fail to raise additional financing as we need it, we will have to
delay or terminate our product development programs.
We expect to continue to spend substantial amounts of capital for our operations
for the foreseeable future. Activities which will require additional
expenditures include:
. research and development programs
. preclinical studies and clinical trials
. regulatory processes
. establishment of commercial scale manufacturing capabilities and
. expansion of sales and marketing activities.
The amount of capital we may need depends on many factors, including:
. The progress, timing and scope of our research and development programs
. The progress, timing and scope of our preclinical studies and clinical
trials
. The time and cost necessary to obtain regulatory approvals
. The time and cost necessary to build our manufacturing facilities and
obtain the necessary regulatory approvals for those facilities
. The time and cost necessary to respond to technological and market
developments
14
. Any changes made or new developments in our existing collaborative,
licensing and other commercial relationships
. Any new collaborative, licensing and other commercial relationships that we
may establish
Moreover, our fixed expenses such as rent, license payments and other
contractual commitments are substantial and will increase in the future. These
fixed expenses will increase because we may enter into:
. additional leases for new facilities and capital equipment
. additional licenses and collaborative agreements
. additional contracts for consulting, maintenance and administrative
services
. additional expenses associated with being a public company.
We believe that the net proceeds of our initial public offering, together with
our available cash, cash equivalents, short-term investment securities and
investment income, will be sufficient to meet our operating and capital
requirements through at least the next 12 months. This estimate is based on
assumptions which may prove to be wrong. As a result, we may need additional
financing prior to that time.
If we fail to obtain regulatory approval to commercially manufacture or sell any
of our future drug products, or if approval is delayed, we will be unable to
generate revenue from the sale of our products.
We must obtain regulatory approval to market our products in the U.S. and
foreign jurisdictions.
We must obtain regulatory approval before marketing or selling our future drug
products. In the United States, we must obtain U.S. Food and Drug Administration
(FDA) approval for each drug that we intend to commercialize. The FDA approval
process is typically lengthy and expensive, and approval is never certain.
Products distributed abroad are also subject to foreign government regulation.
None of our drug products has received regulatory approval to be commercially
marketed and sold. If we fail to obtain regulatory approval we will be unable to
market and sell our future drug products. We have several drug products in
various stages of preclinical and clinical development. Aldurazyme(TM), our
first drug product, is not expected to be commercially available until at least
2000. Our other drug product will not be commercially available for at least
several more years. Because of the risks and uncertainties in biopharmaceutical
development, our drug candidates could take a significantly longer time to gain
regulatory approval than we expect or may never gain approval. If regulatory
approval is delayed our management's credibility, the value of our company and
our operating results may be adversely affected.
To obtain regulatory approval to market our products, preclinical studies and
costly and lengthy clinical trials may be required and the results of the
studies and trials are highly uncertain.
As part of the FDA approval process, we must conduct, at our own expense,
preclinical studies on animals and clinical trials on humans on each drug
candidate. We expect the number of preclinical studies and clinical trials that
the FDA will require will vary depending on the drug product, the disease or
condition the drug is being developed to address and regulations applicable to
the particular drug. We may need to perform multiple preclinical studies using
various doses and formulations before we can begin clinical trials, which could
result in delays in our ability to market any of our drug products.
Furthermore, even if we obtain favorable results in preclinical studies on
animals, the results in humans may be different. After we have conducted
preclinical studies in animals we must demonstrate that our drug products are
safe and effective for use on the target human patients in order to receive
regulatory approval for commercial sale. Adverse or inconclusive clinical
results would stop us from filing for regulatory approval of our products.
Additional factors that can cause delay or termination of our clinical trials
include:
. Slow patient enrollment
. Longer treatment time required to demonstrate efficacy
15
. Lack of sufficient supplies of the drug candidate
. Adverse medical events or side effects in treated patients
. Lack of effectiveness of the drug candidate being tested
Typically, if a drug product is intended to treat a chronic disease safety and
efficacy data must be gathered over an extended period of time which ranges from
six months to three years. In addition, clinical trials on humans are typically
conducted in three phases. The FDA generally requires two pivotal clinical
trials that demonstrate substantial evidence of safety and efficacy and
appropriate dosing in a broad patient population at multiple sites to support an
application for regulatory approval. If a drug is intended for the treatment of
a serious or life-threatening condition and the drug demonstrates the potential
to address unmet medical needs for this condition, a single trial may be
sufficient to prove safety and efficacy under the FDA's Modernization Act of
1997.
Our strategy to conduct only one clinical trial on a small number of patients
for products developed to treat genetic disorders may not be sufficient to
obtain regulatory approval.
We believe that our enzyme drug products will be regulated by the FDA as
biologics rather than drugs because they are manufactured by biological
processes. Our strategy for the development of therapeutics for genetic
disorders is to conduct only one clinical trial on a small number of patients,
which would then be the basis for our submission of a biologics license
application (BLA) to the FDA. For example, at the end of October 1998, we
completed a six-month evaluation of ten patients on our first drug candidate
Aldurazyme(TM). Because 12-month data will be available, the FDA has requested
that we evaluate data for these patients for the 12-month period rather than the
six-month period which formed the basis of our initial evaluation. In addition
the FDA has also requested that we evaluate this data using other criteria that
may demonstrate that the surrogate endpoints are a predictor of clinical
benefit. We are currently performing this evaluation. We cannot assure you that
this evaluation will support our findings with regard to the primary endpoints
in the clinical trial. If this analysis does not support our findings with
regard to the primary endpoints, or if the surrogate endpoints do not predict a
clinical benefit, it could delay the filing of the biologics license application
and could jeopardize FDA approval of Aldurazyme(TM). The FDA may request
additional trials to be conducted. If we have to conduct further clinical
trials, whether for Aldurazyme(TM) or other products we develop in the future,
it would significantly increase our expenses and delay marketing of our product.
Also, the results of initial smaller clinical trials could differ from the
results obtained from subsequent more extensive long-term trials. A significant
difference in the results of multiple clinical trials could cause the FDA to
require still more clinical trials which would significantly delay the approval
process.
The fast track designation for Aldurazyme(TM) may not actually lead to a faster
review process.
Although Aldurazyme(TM) has obtained a fast track designation, we cannot
guarantee a faster review process or faster approval compared to the normal FDA
procedures. If Aldurazyme(TM) is approved, we will be required to conduct a
study after we obtain approval of Aldurazyme(TM) to demonstrate that the primary
endpoints used in our single study are reasonably likely to predict clinical
benefits to the patients. If this post-approval study fails to verify the
clinical benefit of Aldurazyme(TM) or demonstrates that Aldurazyme(TM) is not
safe or effective, our FDA approval can be withdrawn on an expedited basis.
Furthermore, if adverse effects are identified after marketing, FDA approval may
be rapidly revoked and we could not market the drug.
We will not be able to sell our products if we fail to comply with manufacturing
regulations.
Before we can begin commercially manufacturing our products we must obtain
regulatory approval of our manufacturing facility and process. In addition,
manufacture of our drug products must comply with the FDA's current Good
Manufacturing Practices regulations, commonly known as cGMP. The cGMP
regulations govern quality control and documentation policies and procedures.
Our manufacturing facilities are continuously subject to inspection by the FDA,
the State of California and foreign regulatory authorities, before and after
product approval. Because we are currently in the process of developing the
manufacturing site and process for commercial manufacture of Aldurazyme(TM), our
facility has not yet been inspected by any state or federal governmental entity.
We cannot guarantee that BioMarin, or any potential third- party manufacturer of
our drug products, will be able to comply with cGMP regulations. Material
changes to the manufacturing processes after approvals have been granted are
also subject to review and approval by the FDA or other regulatory agencies.
16
We currently have a contract with Harbor-UCLA Research and Education Institute
to manufacture Aldurazyme(TM) in limited quantities for use in preclinical
studies and clinical trials. In order to produce initial commercial requirements
for Aldurazyme(TM) in our facility we will have to prove that the product
manufactured at our facility is comparable to the clinical trial product
produced in the Harbor- UCLA facility. This will require laboratory testing and
may require clinical trials. We must pass FDA and state inspections and
manufacture three process qualification batches to final specifications under
cGMP controls before the Aldurazyme(TM) BLA can be approved. We cannot assure
you that we will pass the inspections in a timely manner, if at all.
If we fail to obtain orphan drug exclusivity for our products, our competitors
may sell products to treat the same conditions and our revenues may be reduced.
As part of our business strategy, we intend to develop drugs that may be
eligible for FDA orphan drug designation. Under the Orphan Drug Act, the FDA may
designate a product as an orphan drug if it is a drug intended to treat a rare
disease or condition, defined as a patient population of less than 200,000. The
company that obtains the first FDA approval for a designated orphan drug for a
given rare disease receives marketing exclusivity for use of that drug for the
stated condition for a period of seven years. However, different drugs can be
approved for the same condition.
Because the extent and scope of patent protection for our drug candidates is
limited, orphan drug designation is particularly important for our products that
are eligible for orphan drug designation. We plan to rely on the exclusivity
period under the orphan drug designation to maintain a competitive position. If
we do not obtain orphan drug exclusivity for any one of our drug products, our
competitors may then sell the same drug to treat the same condition.
We received orphan drug designation from the FDA for Aldurazyme(TM) in September
1997. In February 1999, we received orphan drug designation from the FDA for
BM102. Even if we obtain orphan drug designation, we cannot guarantee that we
will be the first to obtain marketing approval for any orphan indication or that
exclusivity would effectively protect the product from competition. Orphan drug
designation does not shorten the development or FDA review time of a drug so
designated nor give the drug any advantage in the FDA review or approval
process.
Because the target patient populations for our products are small we must
achieve significant market share and obtain high per patient prices for our
products to achieve profitability.
Our initial drug candidates target disorders with small patient populations. As
a result, our prices must be high enough to recover our development costs and
achieve profitability. For example, two of our initial drug products in genetic
disorders, Aldurazyme(TM) and BM102, target patients with MPS-I and MPS-VI,
respectively. We estimate that there are approximately 3,400 patients with MPS-I
and 1,100 patients with MPS-VI in the developed world. We believe that we will
need to market worldwide to achieve significant market share. In addition, we
are developing other drug candidates to treat conditions, such as other genetic
diseases and serious burns, with small patient populations. We cannot be certain
that we will be able to obtain sufficient market share for our drug products at
a price high enough to justify our product development efforts.
If we fail to obtain an adequate level of reimbursement for our drug products by
third-party payors there would be no commercially viable markets for our
products.
The course of treatment for patients with MPS-I using Aldurazyme(TM) is expected
to be expensive. We expect patients to need treatment throughout their
lifetimes. We expect that families of patients will not be capable of paying for
this treatment themselves. There will be no commercially viable market for
Aldurazyme(TM) without reimbursement from third-party payors.
Third-party payors, such as government or private health care insurers,
carefully review and increasingly challenge the price charged for drugs.
Reimbursement rates from private companies vary depending on the third-party
payor, the insurance plan and other factors. Reimbursement systems in
international markets vary significantly by country and by region, and
reimbursement approvals must be obtained on a country-by-country basis. We
cannot be certain that third-party payors will pay for the costs of our drugs
and the courses of treatment. Even if we are able to obtain reimbursement from
third-party payors, we cannot be certain that reimbursement rates will be enough
to allow us to profit from sales of our drugs.
17
We currently have no expertise obtaining reimbursement. We expect to rely on the
expertise of our partner Genzyme to obtain reimbursement for Aldurazyme(TM). We
cannot predict what the reimbursement rates will be. In addition, we will need
to develop our own reimbursement expertise for future drug candidates unless we
enter into collaborations with other companies with the necessary expertise.
We expect that in the future reimbursement will be increasingly restricted both
in the United States and internationally. The escalating cost of health care has
led to increased pressure on the health care industry to reduce costs.
Governmental and private third-party payors have proposed health care reforms
and cost reductions. A number of federal and state proposals to control the cost
of health care, including the cost of drug treatments have been made in the
United States. In some foreign markets, the government controls the pricing
which would affect the profitability of drugs. Current government regulations
and possible future legislation regarding health care may affect our future
revenues from sales of our drugs and may adversely affect our business and
prospects.
If we are unable to protect our proprietary technologywe may not be able to
compete as effectively.
Where appropriate, we seek patent protection for certain aspects of our
technology. Meaningful patent protection may not be available for some of the
enzymes we are developing, including Aldurazyme(TM) and BM102. If we must spend
significant time and money protecting our patents, designing around patents held
by others or licensing, for excessively large fees, patents or other proprietary
rights held by others, our business and prospects may be harmed.
The patent positions of biotechnology companies are extremely complex and
uncertain. The scope and extent of patent protection for some of our products
are particularly uncertain because key information on some of the enzymes we are
developing has existed in the public domain for many years. Other parties have
published the structure of the enzymes, the methods for purifying or producing
the enzymes or the methods of treatment. The composition and genetic sequences
of animal and/or human versions of many of our enzymes, including those for
Aldurazyme(TM) and BM102, have been published and are in the public domain. The
composition and genetic sequences of other MPS enzymes which we intend to
develop as products have also been published. Publication of this information
may prevent us from obtaining composition of matter patents, which are generally
believed to offer the strongest patent protection. For enzymes with no prospect
of composition of matter patents, we will depend on orphan drug status.
In addition, our owned and licensed patents and patent applications do not
ensure the protection of our intellectual property for a number of other
reasons:
. We do not know whether our patent applications will result in actual
patents. For example, we may not have developed a method for treating
a disease before others developed similar methods.
. Competitors may interfere with our patent process in a variety of
ways. Competitors may claim that they invented the claimed invention
prior to us. Competitors may also claim that we are infringing on
their patents and therefore cannot practice our technology as claimed
under our patent. Competitors may also contest our patents by showing
the patent examiner that the invention was not original, novel or was
obvious. As a Company, we have no meaningful experience with
competitors interfering with our patents orpatent applications.
. Even if we receive a patent, it may not provide much practical
protection. If we receive a patent with a narrow scope, then it will
be easier for competitors to design products that do not infringe on
our patent.
. Enforcing patents is expensive and may absorb significant time by our
management. In litigation, a competitor could claim that our issued
patents are not valid for a number of reasons. If the court agrees,
we would lose that patent.
18
In addition, competitors also seek patent protection for their technology. There
are many patents in our field of technology, and we cannot guarantee that we do
not infringe on those patents or that we will not infringe on patents granted in
the future. If a patent holder believes our product infringes on their patent,
the patent holder may sue us even if we have received patent protection for our
technology. If someone else claims we infringe on their technology, we would
face a number of issues, including:
. Defending a lawsuit takes significant time and can be very expensive.
. If the court decides that our product infringes on the competitor's
patent, we may have to pay substantial damages for past infringement.
. The court may prohibit us from selling or licensing the product
unless the patent holder licenses the patent to us. The patent holder
is not required to grant us a license. If a license is available, we
may have to pay substantial royalties or grant cross-licenses to our
patents.
. Redesigning our product so it does not infringe may not be possible
and could require substantial funds and time.
It is also unclear whether our trade secrets will provide useful protection.
While we use reasonable efforts to protect our trade secrets, our employees or
consultants may unintentionally or willfully disclose our information to
competitors. Enforcing a claim that someone else illegally obtained and is using
our trade secrets, like patent litigation, is expensive and time consuming, and
the outcome is unpredictable. In addition, courts outside the United States are
sometimes less willing to protect trade secrets. Our competitors may
independently develop equivalent knowledge, methods and know-how.
We may also support and collaborate in research conducted by government
organizations or by universities. We cannot guarantee that we will be able to
acquire any exclusive rights to technology or products derived from these
collaborations. If we do not obtain required licenses or rights, we could
encounter delays in product development while we attempt to design around other
patents or even be prohibited from developing, manufacturing or selling products
requiring these licenses. There is also a risk that disputes may arise as to the
rights to technology or products developed in collaboration with other parties.
If our joint venture with Genzyme were terminated, we could be barred from
commercializing Aldurazyme(TM) or our ability to commercialize Aldurazyme(TM)
would be delayed.
We are relying on Genzyme to apply the expertise it has developed through the
launch and sale of Ceredase(R) and Cerezyme(R) enzymes for Gaucher disease, a
rare genetic disorder, to the marketing of our initial drug product,
Aldurazyme(TM). Because it is our initial product, our operations are
substantially dependent upon the development of Aldurazyme(TM). We have no
experience selling, marketing or obtaining reimbursement for pharmaceutical
products. In addition, without Genzyme we would be required to pursue foreign
regulatory approvals. We have no experience in seeking foreign regulatory
approvals.
We cannot guarantee that Genzyme will devote the resources necessary to
successfully market Aldurazyme(TM). In addition, either party may terminate the
joint venture for specified reasons, including if the other party is in material
breach of the agreement or has experienced a change of control or has declared
bankruptcy and also is in breach of the agreement. Either party may also
terminate the agreement upon one year prior written notice for any reason after
the earlier of December 31, 2000 or after the joint venture has received the
FDA's approval of the biologics license application for Aldurazyme(TM).
Furthermore, we may terminate the joint venture if Genzyme fails to fulfill its
contractual obligation to pay us $12.1 in cash upon the approval of the
biologics license application for Aldurazyme(TM).
Upon termination of the joint venture one party must buy out the other party's
interest in the joint venture. The party who buys out the other will then also
obtain, exclusively, all rights to Aldurazyme(TM) and any related intellectual
property and regulatory approvals.
19
If the joint venture is terminated by Genzyme for a breach on our part, Genzyme
would be granted, exclusively, all of the rights to Aldurazyme(TM) and any
related intellectual property and regulatory approvals and would be obligated to
buy out our interest in the joint venture. We would then effectively be unable
to develop and commercialize Aldurazyme(TM). If we terminated the joint venture
for a breach by Genzyme, we would be obligated to buy out Genzyme's interest in
the joint venture and, we would then be granted all of these rights to
Aldurazyme(TM) exclusively. While we could then continue to develop
Aldurazyme(TM), that development would be slowed because we would have to divert
substantial capital to buy out Genzyme's interest in the joint venture and would
then have to search for a new partner to commercialize the product and to obtain
foreign regulatory approvals or to develop these capabilities ourselves.
If the joint venture is terminated by us without cause, Genzyme would have the
option, exercisable for one year, to immediately buy out our interest in the
joint venture and obtain all rights to Aldurazyme(TM) exclusively. If the
agreement is terminated by Genzyme without cause, we would have the option,
exercisable for one year, to immediately buy out Genzyme's interest in the joint
venture and obtain these exclusive rights. In event of termination of the buy
out option without exercise by the non-terminating party as described above, all
right and title to Aldurazyme(TM) is to be sold to the highest bidder, with the
proceeds to be split equally between Genzyme and us.
If the joint venture is terminated by us because Genzyme fails to make the $12.1
million payment to us upon FDA approval of the biologics license application for
Aldurazyme(TM), we would be obligated to buy Genzyme's interest in the joint
venture and would obtain all rights to Aldurazyme(TM) exclusively. If the joint
venture is terminated by either party because the other declared bankruptcy and
is also in breach of the agreement, the terminating party would be obligated to
buy out the other and would obtain all rights to Aldurazyme(TM) exclusively. If
the joint venture is terminated by a party because the other party experienced a
change of control, the terminating party shall notify the other party, the
offeree, of its intent to buy out the offeree's interest in the joint venture
for a stated amount set by the terminating party at its discretion. The offeree
must then either accept this offer or agree to buy the terminating party's
interest in the joint venture on those same terms. The party who buys out the
other would then have exclusive rights to Aldurazyme(TM).
We cannot assure you that if the joint venture were terminated and if we were
obligated, or given the option, to buy out Genzyme's interest in the joint
venture, and gain exclusive rights to Aldurazyme(TM), that we will have
sufficient funds to do so or that we will be able to obtain the financing to do
so. If we fail to buy out Genzyme's interest we may be held in breach of the
agreement and may lose any claim to the rights to Aldurazyme(TM) and the related
intellectual property and regulatory approvals. We would then effectively be
prohibited from developing and commercializing the product.
Termination of the joint venture where we retain the rights to Aldurazyme(TM)
could cause us significant delays in product launch in the United States,
difficulties in obtaining third-party reimbursement and delays or failure to
obtain foreign regulatory approval, any of which could hurt our business and
results of operations. Since Genzyme funds 50% of the joint venture's operating
expenses, the termination of the joint venture would double our financial burden
and reduce the funds available to us for other product programs.
If we are unable to manufacture our drug products in sufficient quantities and
at acceptable cost, we may be unable to meet demand for our products and lose
potential revenues.
We have no experience manufacturing drug products in volumes that will be
necessary to support commercial sales. Our unproven manufacturing process may
not meet initial expectations as to schedule, reproducibility, yields, purity,
costs, quality, and other measurements of performance. Improvements in
manufacturing processes typically are very difficult to achieve and are often
very expensive. We cannot know with any certainty how long it might take to make
improvements if it became necessary to do so. If we contract for manufacturing
services with an unproven process, our contractor is subject to the same
uncertainties, high standards and regulatory controls.
20
If we are unable to establish and maintain commercial scale manufacturing within
our planned time and cost parameters, sales of our products and our financial
performance will be adversely affected.
We may encounter problems with any of the following if we attempt to increase
the scale or size of manufacturing:
. Design, construction and qualification of manufacturing facilities
that meet regulatory requirements
. Production yields
. Purity
. Quality control and assurance
. Shortages of qualified personnel
. Compliance with FDA regulations
We are developing a total of 43,000 square feet of space at two facilities, one
in Novato and one in Torrance, for the manufacture of Aldurazyme(TM). The
construction and qualification of these facilities may take longer than planned
and the actual construction costs of these facilities may be higher than those
which we have budgeted. We expect that the manufacturing process of all of our
new products, including BM102, will also require lengthy development time before
we can begin manufacturing them in commercial quantity. Even if we can establish
this capacity, we cannot be certain that manufacturing costs will be
commercially reasonable, especially if reimbursement is substantially lower than
expected.
In order to achieve our product cost targets we must develop efficient
manufacturing processes either by
. improving the colonies of cells which have a common genetic make-up,
or cell lines,
. improving the processes licensed from others, or
. developing a recombinant cell line and production processes.
A recombinant cell line is a cell line with foreign DNA inserted which is used
to produce a foreign protein that it would not have otherwise produced. The
development of a stable, high production cell line for any given enzyme is
risky, expensive and unpredictable and may not yield adequate results. In
addition, the development of protein purification processes is difficult and may
not produce the high purity required with acceptable yield and costs. If we are
not able to develop efficient manufacturing processes, the investment in
manufacturing capacity sufficient to satisfy market demand will be much greater
and will place heavy financial demands upon us. If we do not achieve our
manufacturing cost targets, we will have lower margins and reduced profitability
in commercial production and greater losses in manufacturing start-up phases.
If we are unable to increase our marketing and distribution capabilities or to
enter into agreements with third parties to do so, our ability to generate
revenues will be diminished.
If we cannot increase our marketing capabilities either by developing our sales
and marketing organization or by entering into agreements with others, we may be
unable to successfully sell our products. If we are unable to effectively sell
our drug products, our ability to generate revenues will be diminished.
To increase our distribution and marketing for both our drug candidates and our
Glyko, Inc. products, we will have to increase our current sales force and/or
enter into third-party marketing and distribution agreements. We cannot
guarantee that we will be able to hire in a timely manner, the qualified sales
and marketing personnel we need if at all. Nor can we guarantee that we will be
able to enter into any marketing or distribution agreements on acceptable terms,
if at all. If we cannot increase our marketing capabilities as we intend, either
by increasing our sales force or entering into agreements with third parties,
sales of our products may be adversely affected.
21
We have entered into a joint venture with Genzyme where Genzyme will be
responsible for marketing and distributing Aldurazyme(TM). We cannot guarantee
that we will be able to establish sales and distribution capabilities or that
BioMarin, the joint venture or any future collaborators will successfully sell
any of our drug candidates.
If we fail to compete successfully, our revenues and operating results will be
adversely affected.
Our competitors may develop, manufacture and market products that are more
effective or less expensive than ours. They may also obtain regulatory approvals
for their products faster than we can obtain them, including orphan drug
designation, or commercialize their products before we do. If our competitors
successfully commercialize a product which treats a given rare genetic disease
before we do, we will effectively be precluded from developing a product to
treat that disease because the patient populations of the rare genetic diseases
are so small. These companies also compete with us to attract qualified
personnel and parties for acquisitions, joint ventures or other collaborations.
They also compete with us to attract academic research institutions as partners
and to license these institution's proprietary technology. If our competitors
successfully enter into partnering arrangements or license agreements with
academic research institutions, we will then be precluded from pursuing those
specific opportunities. Since each of these opportunities is unique, we may not
be able to find a substitute. Several pharmaceutical and biotechnology companies
have already established themselves in the field of enzyme therapeutics,
including Genzyme, our joint venture partner. These companies have already begun
many drug development programs, some of which may target diseases that we are
also targeting, and have already entered into partnering and licensing
arrangements with academic research institutions, reducing the pool of available
opportunities.
Universities and public and private research institutions are also competitors.
While these organizations primarily have educational objectives, they may
develop proprietary technology and acquire patents that we may need for the
development of our drug products. We will attempt to license this proprietary
technology, if available. These licenses may not be available to us on
acceptable terms, if at all. We also directly compete with a number of these
organizations to recruit personnel, especially scientists and technicians.
We believe that established technologies provided by other companies, such as
laboratory and testing services firms compete with Glyko Inc.'s products and
services. For example, Glyko, Inc.'s FACE Imaging System competes with
alternative carbohydrate analytical technologies, including capillary
electrophoresis, high-pressure liquid chromatography, mass spectrometry and
nuclear magnetic resonance spectrometry. These competitive technologies have
established customer bases and are more widely used and accepted by scientific
and technical personnel because they can be used for non-carbohydrate
applications. Companies competing with Glyko, Inc. may have greater financial,
manufacturing and marketing resources and experience.
If we fail to manage our growth or fail to recruit and retain personnel, our
product development programs may be delayed.
Our rapid growth has strained our managerial, operational, financial and other
resources. We expect this growth to continue. We recently entered into a joint
venture with Genzyme. If we receive FDA approval to market Aldurazyme(TM), the
joint venture will be required to devote additional resources to support the
commercialization of Aldurazyme(TM).
To manage expansion effectively, we need to continue to develop and improve our
operating and financial systems, sales and marketing capabilities. We cannot
guarantee that our systems, procedures or controls will be adequate to support
our operations or that our management will be able to manage successfully future
market opportunities or our relationships with customers and other third
parties.
Our future growth and success depend on our ability to recruit, retain, manage
and motivate our employees. The loss of key scientific, technical and managerial
personnel may delay our product development programs. Any harm to our research
and development programs would harm our business and prospects.
22
Because of the specialized scientific nature of our business, we rely heavily on
our ability to attract and retain qualified scientific, technical and managerial
personnel. In particular, the loss of Grant W. Denison, Jr., Chairman and Chief
Executive Officer, John C. Klock, M.D., President and Secretary or Christopher
M. Starr, Ph.D., Vice President for Research and Development would be
detrimental to us. While each of these individuals is party to an employment
agreement with us, which includes financial incentives for each of them to
remain employed with us, these agreements each terminate in June 2000 and we
cannot guarantee that any of them will remain employed with us beyond that time.
In addition, these agreements do not restrict their ability to compete with us
after their employment is terminated. The competition for qualified personnel in
the biopharmaceutical field is intense. We cannot be certain that we will
continue to attract and retain qualified personnel necessary for the development
of our business.
If product liability lawsuits are successfully brought against us, we may incur
substantial liabilities.
We are exposed to the potential product liability risks inherent in the testing,
manufacturing and marketing of human drug treatments. We currently do not
maintain insurance against product liability lawsuits. The joint venture with
Genzyme maintains clinical liability insurance for Aldurazyme(TM) which covers
clinical trials of that product, since their inception. Although we intend to
obtain product liability insurance before our clinical trials of BM102 and
shortly before initiating clinical trials for our other products, we cannot be
certain that we will be able to obtain adequate insurance coverage. In addition,
we may be subject to claims in connection with our current clinical trials for
Aldurazyme(TM) for which current insurance coverage is not adequate. We cannot
be certain that if Aldurazyme(TM) receives FDA approval, the product liability
insurance the joint venture will need to obtain in connection with the
commercial sales of Aldurazyme(TM) will be available at a reasonable cost. In
addition, we cannot be certain that we can successfully defend any product
liability lawsuit brought against us. If we are the subject of a successful
product liability claim which exceeds the limits of any insurance coverage we
may obtain, we may incur substantial liabilities which would adversely affect
our earnings and financial condition.
If we experience any problems with Year 2000 compliance our operations may be
disrupted.
The following is intended to constitute "Year 2000 Readiness Disclosure" under
the Year 2000 Information and Readiness Disclosure Act of 1998.
Beginning in the year 2000, the date fields coded in certain software products
and computer systems will need to accept four digit entries in order to
distinguish 21st century dates from 20th century dates (commonly known as the
year 2000 problem). It is not clear what potential problems may arise as the
biopharmaceutical industry, and other industries, try to resolve this year 2000
problem.
It is possible that our currently installed computer systems, software products
or other business systems, or those of our suppliers or service providers,
working either alone or in conjunction with other software or systems, will not
accept input of, store, manipulate and output dates for the years 1999, 2000 or
subsequent years without error or interruption. We have formed a team to review
and resolve those aspects of the year 2000 problem that are within our direct
control and adjust to or influence those aspects that are not within our direct
control. The team has reviewed our software products, including those under
development, and determined that our software products do not use date data and
are year 2000 compliant. Our biopharmaceutical products do not have any year
2000 exposure. Based on representations from our vendors, the team has reviewed
the year 2000 compliance status of our major internal information technology
programs and systems used for administrative requirements and determined that
they are year 2000 compliant.
Some risks associated with the year 2000 problem are beyond our ability to
control, including the extent to which our suppliers and service providers can
address the year 2000 problem. The failure by a third party to adequately
address the year 2000 issue could have an adverse effect on their operations,
which could have an adverse effect on us. We are assessing the possible effects
on our operations of the possible failure of our key suppliers and providers,
contractors and collaborators to identify and remedy, if practical, potential
year 2000 problems.
23
Our stock price may be volatile and your investment in our stock could suffer a
decline in value.
Our valuation and the stock price in the period since the IPO have had no
meaningful relationship to current or historical earnings, asset values, book
value or any other criteria based on historical values. The market price of the
common stock will fluctuate and may be higher or lower in the future due to
factors including:
. Progress of Aldurazyme(TM) and our other lead drug candidates through
worldwide regulatory processes, especially Aldurazyme(TM)
regulatory actions in the United States
. Results of clinical trials, announcements of technological
innovations or new products by us or our competitors
. Government regulatory action affecting our drug candidates or our
competitors' drug candidates in both the United States and foreign
countries
. Developments or disputes concerning patent or proprietary rights
. General market conditions for emerging growth and biopharmaceutical
companies
. Economic conditions in the United States or broad
. Actual or anticipated fluctuations in our operating results
. Broad market fluctuations may cause the market price of our common
stock to fluctuate
. Changes in financial or business estimates by securities analysts
In addition, the value of our common stock may fluctuate because it is listed on
both the Nasdaq National Market and the Swiss Exchange New Market. Because we
have accumulated relatively limited experience since July 23, 1999, in observing
the trading of our stock on two markets, we cannot be certain what effect, if
any, the dual listing will have on the future price of our stock in either
market. Under different conditions in the future, listing on both exchanges may
increase stock price volatility due to:
. trading in different time zones
. different ability to buy or sell our stock
. different trading volume
In the past, following periods of large price declines in the public market
price of a company's securities, securities class action litigation has often
been initiated against that company. Litigation of this type could result in
substantial costs and diversion of management's attention and resources, which
would hurt our business. Any adverse determination in litigation could also
subject us to significant liabilities.
If our officers, directors and largest stockholder elect to act together they
may be able to control our management and operations, acting in their best
interests and not necessarily those of other stockholders.
24
Our directors and officers control approximately 17.4% of the outstanding shares
of our common stock. GBL owns 32.7% of the outstanding shares of capital stock.
Three of six GBL directors are officers or directors of BioMarin. As a result,
due to their concentration of stock ownership, directors and officers, together
with GBL if they act together, may be able to otherwise control our management
and operations, and may be able to prevail on all matters requiring a
stockholder vote including:
. The election of all directors
. The amendment of charter documents or the approval of a merger, sale
of assets or other major corporate transactions
. The defeat of any non-negotiated takeover attempt that might
otherwise benefit the public stockholders
Anti-takeover provisions in our charter documents and under Delaware law may
make an acquisition of us, which may be beneficial to our stockholders, more
difficult.
BioMarin is incorporated in Delaware. Certain anti-takeover provisions of
Delaware law and our charter documents may make a change in control of BioMarin
more difficult, even if a change in control would be beneficial to the
stockholders. Our anti-takeover provisions include provisions in the certificate
of incorporation providing that stockholders' meetings may only be called by the
board of directors and a provision in the bylaws providing that the stockholders
may not take action by written consent. Additionally, our board of directors
have the authority to issue 1,000,000 shares of preferred stock and to determine
the terms of those shares of stock without any further action by the
stockholders. The rights of holders of our common stock are subject to the
rights of the holders of any preferred stock that may be issued. The issuance of
preferred stock, could make it more difficult for a third party to acquire a
majority of the outstanding voting stock of BioMarin. Delaware law also
prohibits corporations from engaging in a business combination with any holders
of 15% or more of their capital stock until the holder has held the stock for
three years unless, among other possibilities, the board of directors approves
the transaction. The board of directors may use these provisions to prevent
changes in the management and control of our company. Also, under applicable
Delaware law, our board of directors may adopt additional anti-takeover measures
in the future.
Item 3. Quantitative and Qualitative Disclosure about
Market Risk.
The Company's exposure to market risk for changes in interest rates relates
primarily to the Company's investment portfolio. The Company places its
investments with high credit issuers and by policy limits the amount of credit
exposure to any one issuer. As stated in its policy, the Company will seek to
improve the safety and likelihood of preservation of its invested funds by
limiting default risk and market risk. The Company has no investments
denominated in foreign country currencies and therefore is not subject to
foreign exchange risk.
The Company mitigates default risk by investing in high credit quality
securities and by positioning its portfolio to respond appropriately to a
significant reduction in a credit rating of any investment issuer or guarantor.
The portfolio includes only marketable securities with active secondary or
resale markets to ensure portfolio liquidity.
The table below presents the carrying value for the Company's investment
portfolio. The carrying value approximates fair value at September 30, 1999.
Investment portfolio:
Carrying value(in $ thousands)
Cash and cash equivalents $32,585
Short-term investments 39,307*
Certificates of deposit 265
Total $ 72,157
* 100% in United States agency securities.
25
PART II. OTHER INFORMATION
Item 1. Legal Proceedings. None.
Item 2. Changes in Securities and Uses of Proceeds.
ISSUANCES OF UNREGISTERED SECURITIES
During the period covered by this report, registrant issued the following
unregistered securities:
From July 1 to September 30, 1999, registrant granted options to purchase
373,500 shares of common stock to officers, directors, employees and consultants
in reliance on Rule 701 promulgated under the Securities Act or in reliance on
the exemption from the regulation requirements provided by Section 4(2) of the
Securities Act.
During the period from July 1, to September 30, 1999, options to purchase 10,822
shares of common stock were exercised in reliance on Rule 701 promulgated under
the Securities Act or in reliance on the exemption from the registration
requirements provided by Section 4(2) of the Securities Act.
In a private placement concurrent with the IPO, Genzyme invested in the Company
$10 million at the IPO price of $13 per share (769,230 shares of common stock)
in reliance on the exemption from registration requirements provided by
Regulation D promulgated under the Securities Act.
USES OF PROCEEDS
The effective date of registrant's Registration Statement on Form S-1 was July
22, 1999. The offering commenced on July 23, 1999 and concluded on August 25,
1999. All of the securities registered under the Registration Statement were
sold in the offering. The global co-coordinators of the offering were Bank J.
Vontobel & Co. AG and U.S. Bancorp Piper Jaffray Inc. The Registration Statement
covered the sale of 4,500,000 shares of registrant's Common Stock, par value
$0.001 per share, as well as the sale of an additional 675,000 shares of
registrant's Common Stock upon the exercise by the underwriters of their
over-allotment option. The aggregate offering price of the number of shares
registered in the offering was $67,275,000. A total of 5,175,000 shares were
sold in the offering, the aggregate price of which was $67,275,000.
As of November 1, 1999, the expenses incurred by registrant in connection with
the issuance and distribution of the securities registered (including
underwriters' discounts and commissions) were $7.3 million. No direct or
indirect payments for these expenses were made to registrant's directors,
officers or persons owning 10% or more of registrant's outstanding equity
securities. After deducting these expenses, registrant's net proceeds from the
offering were $60.0 million.
Item 3. Defaults upon Senior Securities. None.
Item 4. Submission of Matters to a Vote of Security Holders. None.
Item 5. Other Information. None.
26
Item 6. Exhibits and Reports on Form 8-K.
(a) The following documents are filed as part of this report
See Exhibit Index attached hereto.
(b) Reports on Form 8K
No reports were filed on Form 8-K during the three months
ended September 30, 1999.
27
SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934,
the registrant caused this Report to be signed on its behalf by the
undersigned, thereunto duly authorized.
BIOMARIN PHARMACEUTICAL INC.
Dated: November 12, 1999 By: \s\ Raymond W. Anderson
- ---------------------------------------- ------------------------------
Raymond W. Anderson
Chief Financial Officer and
V.P. Finance and Administration
28
EXHIBIT INDEX
Exhibit
Number Description of Document
- -------- -------------------------------
1.1 Form of Underwriting Agreement is incorporated herein by reference to
the Registration Statement on Form S-1 filed on July 21, 1999.
2.1 Share Exchange Agreement with Glyko Biomedical, Ltd. is incorporated
herein by reference to the Registration Statement on Form S-1 filed on
May 4, 1999.
3.1A Amended and Restated Certificate of Incorporation of BioMarin
Pharmaceutical Inc., a Delaware Corporation, as filed on March 22,
1999 is incorporated herein by reference to the Registration Statement
on Form S-1 filed on May 4, 1999.
3.1B(1) Form of Amended and Restated Certificate of Incorporation of
BioMarin Pharmaceutical Inc., a Delaware Corporation is incorporated
herein by reference to the Registration Statement on Form S-1 filed on
July 6, 1999.
3.2 Amended and Restated Bylaws of BioMarin Pharmaceutical Inc., a
Delaware corporation is incorporated herein by reference to the
Registration Statement on Form S-1 filed on July 6, 1999.
4.1 Form of Amended and Restated Registration Rights Agreement, by and
among the Company and the investors named therein is incorporated
herein by reference to the Registration Statement on Form S-1 filed on
May 4, 1999.
5.1 Opinion of Wilson Sonsini Goodrich & Rosati is incorporated herein by
reference to the Registration Statement on Form S-1 filed on July 21,
1999.
10.1 Form of Indemnification Agreement for directors and officers is
incorporated herein by reference to the Registration Statement on Form
S-1 filed on May 4, 1999.
10.2 1997 Stock Plan, as amended on December 22, 1998, and forms of
agreements thereunder is incorporated herein by reference to the
Registration Statement on Form S-1 filed on May 4, 1999.
10.3 1998 Director Option Plan and forms of agreements thereunder is
incorporated herein by reference to the Registration Statement on Form
S-1 filed on May 4, 1999.
10.4 1998 Employee Stock Purchase Plan and forms of agreements thereunder
is incorporated herein by reference to the Registration Statement on
Form S-1 filed on May 4, 1999.
10.5 Amended and Restated Founder's Stock Purchase Agreement with Dr. John
C. Klock dated as of October 1, 1997 with exhibits is incorporated
herein by reference to the Registration Statement on Form S-1 filed on
May 4, 1999.
10.6 Amended and Restated Founder's Stock Purchase Agreement with Grant W.
Denison, Jr. dated as of October 1, 1997 with exhibits is incorporated
herein by reference to the Registration Statement on Form S-1 filed on
May 4, 1999.
10.7 Amended and Restated Founder's Stock Purchase Agreement with Dr.
Christopher M. Starr dated as of October 1, 1997 with exhibits is
incorporated herein by reference to the Registration Statement on Form
S- 1 filed on May 4, 1999.
10.8 Employment Agreement with Dr. John C. Klock dated June 26, 1997, as
amended is incorporated herein by reference to the Registration
Statement on Form S-1 filed on June 14, 1999.
10.9 Employment Agreement with Grant W. Denison, Jr. dated June 26, 1997,
as amended is incorporated herein by reference to the Registration
Statement on Form S-1 filed on May 4, 1999.
10.10 Employment Agreement with Dr. Christopher M. Starr dated June 26,
1997, as amended is incorporated herein by reference to the
Registration Statement on Form S-1 filed on May 4, 1999.
10.11 Employment Agreement with Raymond W. Anderson dated June 22, 1998, as
amended is incorporated herein by reference to the Registration
Statement on Form S-1 filed on May 4, 1999.
10.12 Employment Agreement with Stuart J. Swiedler, M.D., Ph.D., dated May
29, 1998, as amended is incorporated herein by reference to the
Registration Statement on Form S-1 filed on May 4, 1999.
29
Exhibit
Number Description of Document
- -------- -------------------------------
10.13 Employment Agreement with Emil Kakkis, M.D., Ph.D., dated June 30,
1998, as amended is incorporated herein by reference to the
Registration Statement on Form S-1 filed on May 4, 1999.
10.14 Employment Agreement between Brian K. Brandley, Ph.D and Glyko, Inc.
dated February 22, 1998, as amended is incorporated herein by
reference to the Registration Statement on Form S-1 filed on May 4,
1999.
10.15 License Agreement with Glyko Biomedical, Ltd. dated June 26, 1997 with
exhibits attached is incorporated herein by reference to the
Registration Statement on Form S-1 filed on May 4 1999.
10.16(2) Option Agreement with W.R. Grace & Co. dated as of May 1, 1998 is
incorporated herein by reference to the Registration Statement on Form
S-1 filed on June 14, 1999.
10.17(2) Grant Terms and Conditions Agreement with Harbor-UCLA Research and
Education Institute dated April 1, 1997, as amended is incorporated
herein by reference to the Registration Statement on Form S-1 filed on
June 14, 1999.
10.18(2) License Agreement with Women's and Children's Hospital, Adelaide,
Australia dated August 14, 1998 is incorporated herein by reference to
the Registration Statement on Form S-1 filed on July 21, 1999.
10.19 Lease Agreement dated May 18, 1998 for 371 Bel Marin Keys Boulevard,
as amended is incorporated herein by reference to the Registration
Statement on Form S-1 filed on May 4, 1999.
10.20 Standard NNN Lease dated June 25, 1998 for 46 Galli Drive is
incorporated herein by reference to the Registration Statement on Form
S-1 filed on May 4, 1999.
10.21 Standard Industrial Commercial Single-Tenant Lease dated May 29, 1998
for 110 Digital Drive, as amended is incorporated herein by reference
to the Registration Statement on Form S-1 filed on May 4, 1999.
10.22 Sublease dated June 24, 1998 for 1123 West Carson Street is
incorporated herein by reference to the Registration Statement on Form
S-1 filed on May 4, 1999.
10.23 Commercial Lease and Deposit Receipt with Glyko, Inc. for 11 Pimentel
Court and 13 Pimentel Court, dated December 23, 1996 is incorporated
herein by reference to the Registration Statement on Form S-1 filed on
May 4, 1999.
10.24 Collaboration Agreement with Genzyme Corporation dated September 4,
1998 is incorporated herein by reference to the Registration Statement
on Form S-1 filed on July 21, 1999.
10.25 Purchase Agreement with Genzyme Corporation dated September 4, 1998 is
incorporated herein by reference to the Registration Statement on Form
S-1 filed on May 4, 1999.
10.26 Subscription Agreement with Genzyme dated September 4, 1998 is
incorporated herein by reference to the Registration Statement on Form
S-1 filed on May 4, 1999.
10.27 Form of Convertible Note Purchase Agreement dated as of April 12, 1999
with form of convertible promissory note is incorporated herein by
reference to the Registration Statement on Form S-1 filed on May 4,
1999.
10.28 Astro License Agreement dated December 18, 1990 among Glyko, Inc.,
Astromed, Ltd., and Astroscan, Ltd. is incorporated herein by
reference to the Registration Statement on Form S-1 filed on June 14,
1999.
10.29 Glycomed License Agreement dated December 18, 1990 between Glyko,
Inc., and Glycomed, Inc. is incorporated herein by reference to the
Registration Statement on Form S-1 filed on June 14, 1999.
10.30 Operating Agreement with Genzyme Corporation is incorporated herein by
reference to the Registration Statement on Form S-1 filed on July 6,
1999.
21.1 List of Subsidiaries is incorporated herein by reference to the
Registration Statement on Form S-1 filed on May 4, 1999.
23.1 Consent of Independent Public Accountants is incorporated herein by
reference to the Registration Statement on Form S-1 filed on July 21,
1999.
23.2 Consent of Counsel (included in Exhibit 5.1) is incorporated herein by
reference to the Registration Statement on Form S-1 filed on July 21,
1999.
24.1 Power of Attorney is incorporated herein by reference to the
Registration Statement on Form S-1 filed on May 4, 1999.
27.1 Financial Data Schedule (available in EDGAR format only).
30