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| STEM > SEC Filings for STEM > Form 10-Q on 5-Nov-2009 | All Recent SEC Filings |
5-Nov-2009
Quarterly Report
This report contains forward looking statements within the meaning of
Section 27A of the Securities Act and Section 21E of the Securities Exchange Act
that involve substantial risks and uncertainties. Such statements include,
without limitation, all statements as to expectation or belief and statements as
to our future results of operations; the progress of our research, product
development and clinical programs; the need for, and timing of, additional
capital and capital expenditures; partnering prospects; costs of manufacture of
products; the protection of, and the need for, additional intellectual property
rights; effects of regulations; the need for additional facilities; and
potential market opportunities. Our actual results may vary materially from
those contained in such forward-looking statements because of risks to which we
are subject, including the fact that additional trials will be required to
confirm the safety and demonstrate the efficacy of the Company's HuCNS-SC cells
for the treatment of neuronal ceroid lipofuscinosis (NCL, also known as Batten
disease) or any other disease; uncertainty as to whether the U.S. Food and Drug
Administration (FDA) or other regulatory authorities will permit us to proceed
with clinical testing of proposed products despite the novel and unproven nature
of our technologies; the risk that one or more of our clinical trials or studies
could be substantially delayed beyond their expected dates or cause us to incur
substantial unanticipated costs; uncertainties in our ability to obtain the
capital resources needed to continue our current research and development
operations and to conduct the research, preclinical development and clinical
trials necessary for regulatory approvals; uncertainties about the design of
future clinical trials and whether the Company will receive the necessary
support of a clinical trial site and its institutional review board to pursue
future clinical trials in NCL, PMD or in proposed therapies for other diseases
or conditions; the uncertainty regarding our ability to obtain a corporate
partner or partners, if needed, to support the development and commercialization
of our potential cell-based therapeutics products; the uncertainty regarding the
outcome of our clinical trials or studies we may conduct in the future; the
uncertainty regarding the validity and enforceability of our issued patents; the
risk that we may not be able to manufacture additional master and working cell
banks when needed; the uncertainty whether any products that may be generated in
our cell-based therapeutics programs will prove clinically safe and effective;
the uncertainty whether we will achieve significant revenue from product sales
or become profitable; uncertainties regarding our obligations with respect to
our former encapsulated cell therapy facilities in Rhode Island; obsolescence of
our technologies; competition from third parties; intellectual property rights
of third parties; litigation risks; and other risks to which we are subject. All
forward-looking statements attributable to us or to persons acting on our behalf
are expressly qualified in their entirety by the cautionary statements and risk
factors set forth in "Risk Factors" in Part II, Item 1A of this report and
Part I, Item 1A included in our Annual Report on Form 10-K for the fiscal year
ended December 31, 2008.
Overview
The Company
We are focused on developing and commercializing cell-based technologies. Our
research and development (R&D) programs are primarily focused on identifying and
developing potential cell-based therapeutics which can either restore or support
organ function. In particular, since we relocated our corporate headquarters to
California in 1999, our R&D efforts have been directed at refining our methods
for identifying, isolating, culturing, and purifying the human neural stem cell
and human liver engrafting cells (hLEC) and developing these as potential
cell-based therapeutics for the central nervous system (CNS) and the liver,
respectively. In our CNS Program, our HuCNS-SCŪ product candidate (purified
human neural stem cells) is in clinical development for two indications. In
January 2009, we completed a six patient Phase I clinical trial of HuCNS-SC
cells in infantile and late infantile neuronal ceroid lipofuscinosis (NCL), two
forms of a group of disorders often referred to as Batten disease. The data from
this Phase I trial showed that the HuCNS-SC cells were well tolerated, and there
was evidence that the donor cells engrafted and survived. In December 2008, the
FDA approved our IND to initiate a Phase I clinical trial of HuCNS-SC cells in a
second indication, Pelizeaus-Merzbacher Disease (PMD), a fatal myelination
disorder in the brain. In September 2009, we announced that we plan to initiate
the PMD trial at University of California-San Francisco Children's Hospital. We
expect to begin enrolling patients by the end of 2009 and that the trial will
take 12-18 months to complete. In addition, our HuCNS-SC cells are in
preclinical development for spinal cord injury and retinal disorders. In our
Liver Program, we are in preclinical research and development with our human
liver engrafting cells and we plan to seek the necessary approvals to initiate a
clinical study to evaluate hLEC as a potential cellular therapy, with the
initial indication likely to be liver-based metabolic disorders. For a brief
description of our significant therapeutic research and development programs see
Overview "Research and Development Programs" in the Business Section of Part I,
Item 1 included in our Annual Report on Form 10-K for the fiscal year ended
December 31, 2008. We have also conducted research on several other cell types
and in other areas, which could lead to other possible product candidates,
process improvements or further research activities.
On April 1, 2009, we acquired the operations of Stem Cell Sciences Plc (SCS).
The acquired business includes proprietary cell technologies relating to
embryonic stem cells, induced pluripotent stem (iPS) cells, and tissue-derived
(adult) stem cells; expertise and infrastructure for providing cell-based assays
for drug discovery; a media formulation and reagent business; and an
intellectual property portfolio with claims relevant to cell processing,
reprogramming and manipulation, as well as to gene targeting and insertion.
These acquired operations will help us pursue applications of our cell
technologies to develop cell-based research tools, which we believe represent
nearer-term commercial opportunities. See Note 9 "Acquisition of SCS Operations"
in the notes to condensed financial statements of Part I, Item 1 of this form
10-Q for further information.
We have not derived any revenue or cash flows from the sale or
commercialization of any therapeutic products. Through our acquisition of the
SCS operations, we have derived revenue from sales and royalties on sales of
cell culture media. We have also derived revenue from licensing rights to our
intellectual property. To date, all such revenue has been limited and there can
be no assurance that these revenues will increase. As a result, we have incurred
annual operating losses since inception and expect to incur substantial
operating losses in the future. Therefore, we are dependent upon external
financing from equity and debt offerings and revenue from collaborative research
arrangements with corporate sponsors to finance our operations. We have no such
collaborative research arrangements at this time and there can be no assurance
that such financing or partnering revenue will be available when needed or on
terms acceptable to us.
Before we can derive revenue or cash inflows from the commercialization of
any of our therapeutic product candidates, we will need to: (i) conduct
substantial in vitro testing and characterization of our proprietary cell types,
(ii) undertake preclinical and clinical testing for specific disease
indications; (iii) develop, validate and scale-up manufacturing processes to
produce these cell-based therapeutics, and (iv) pursue required regulatory
approvals. These steps are risky, expensive and time consuming.
Overall, we expect our R&D expenses to be substantial and to increase for the
foreseeable future as we continue the development and clinical investigation of
our current and future therapeutic product candidates. In addition, we expect
our expenses and expenditures to increase as we begin to develop and
commercialize non-therapeutic applications of our cell-based technologies.
However, expenditures on R&D programs are subject to many uncertainties,
including whether we develop our product candidates with a partner or
independently. We cannot forecast with any degree of certainty which of our
product candidates or technologies will be subject to future collaboration, when
such collaboration agreements will be secured, if at all, and to what degree
such arrangements would affect our development plans and capital requirements.
In addition, there are numerous factors associated with the successful
commercialization of any of our cell-based products, including future regulatory
requirements and legal restrictions on the procurement of human tissue for
medical research, many of which cannot be determined with accuracy at this time
given the stage of
our development and the novel nature of stem cell technologies. The regulatory
pathways, both in the United States and internationally, are complex and fluid
given the novel and, in general, clinically unproven nature of stem cell
technologies. At this time, due to such uncertainties and inherent risks, we
cannot estimate in a meaningful way the duration of, or the costs to complete,
our R&D programs or whether, when or to what extent we will generate revenues or
cash inflows from the commercialization and sale of any of our therapeutic
product candidates or any non-therapeutic applications of our cell-based
technologies. While we are currently focused on advancing each of our product
development programs, our future R&D expenses will depend on the determinations
we make as to the scientific and clinical prospects of each product candidate,
as well as our ongoing assessment of the regulatory requirements and each
product candidate's commercial potential.
There can be no assurance that we will be able to develop any product
successfully, or that we will be able to recover our development costs, whether
upon commercialization of a developed product or otherwise. We cannot provide
assurance that any of these programs will result in products that can be
marketed or marketed profitably. If certain of our development-stage programs do
not result in commercially viable products, our results of operations could be
materially adversely affected.
Significant Events
In November 2009, we raised gross proceeds of $12,500,000 through the sale to
certain institutional investors of 10,000,000 shares of common stock and
warrants to purchase 4,000,000 shares of common stock. The common stock and
warrants were sold in units, with each unit consisting of (i) one share of
common stock and (ii) a warrant to purchase 0.4 of a share of common stock at an
exercise price of $1.50 per share, and the purchase price was $1.25 per unit.
The warrants will generally be exercisable for a period of five years beginning
six months after the date of issuance. The shares and warrants were offered as a
registered direct offering under our effective shelf registration statement
previously filed with the SEC. We received total proceeds, net of offering
expenses and placement agency fees, of approximately $11,900,000. The net
proceeds of the financing will be used for general corporate purposes, including
working capital, product development and capital expenditures, as well as for
other strategic purposes.
In October 2009, we announced new preclinical data showing that our human
neural stem cells protect cone photoreceptors (cones) in the eye from
progressive degeneration and preserve visual function long term. Cones are light
sensing cells that are highly concentrated within the macula of the human eye,
and the ability to protect these cells suggests a promising approach to treating
age-related macular degeneration (AMD), the leading cause of vision loss and
blindness in people over the age of 55. These findings were presented at the
Society for Neuroscience 2009 Annual Meeting.
In September 2009, we announced the publication of preclinical data
demonstrating for the first time that transplantation of our proprietary,
purified human neural stem cells delays the loss of motor function in a mouse
model of infantile neuronal ceroid lipofuscinosis (NCL). NCL, commonly referred
to as Batten disease, is a fatal neurodegenerative disorder in children. This
paper, "Neuroprotection of Host Cells by Human Central Nervous System Stem Cells
in a Mouse Model of Infantile Neuronal Ceroid Lipofuscinosis," was featured in
the September 2009 edition of the peer-reviewed journal Cell Stem Cell.
In September 2009, we announced that we plan to initiate with the University
of California, San Francisco (UCSF) Children's Hospital a Phase I clinical trial
to evaluate the therapeutic potential of our proprietary HuCNS-SCŪ product
candidate (purified human neural stem cells) to treat Pelizaeus-Merzbacher
Disease (PMD), a myelination disorder that primarily affects infants and young
children. In this trial, patients with a fatal form of PMD will be transplanted
with our HuCNS-SC cells to evaluate safety and to explore the ability of the
cells to myelinate the patients' nerve axons.
Critical Accounting Policies and the Use of Estimates
The accompanying discussion and analysis of our financial condition and
results of operations are based on our condensed consolidated financial
statements and the related disclosures, which have been prepared in accordance
with U.S. GAAP. The preparation of these condensed consolidated financial
statements requires management to make estimates, assumptions, and judgments
that affect the reported amounts in our condensed consolidated financial
statements and accompanying notes. These estimates form the basis for making
judgments about the carrying values of assets and liabilities. We base our
estimates and judgments on historical experience and on various other
assumptions that we believe to be reasonable under the circumstances, and we
have established internal controls related to the preparation of these
estimates. Actual results and the timing of the results could differ materially
from these estimates.
Stock-Based Compensation
U.S. GAAP requires us to recognize expense related to the fair value of our
stock-based payment awards, including employee stock options. Under the
provisions of U.S. GAAP, employee stock-based payment is estimated at the date
of grant based on the award's fair value using the Black-Scholes-Merton
(Black-Scholes) option-pricing model and is recognized as expense ratably over
the requisite service period. The Black-Scholes option-pricing model requires
the use of certain assumptions, the most significant of which are our estimates
of the expected volatility of the market price of our stock and the expected
term of the award. Our estimate of the expected volatility is based on
historical volatility. The expected term represents the period during which our
stock-based awards are expected to be outstanding. We estimate the expected term
based on historical experience of similar awards, giving consideration to the
contractual terms of the awards, vesting requirements, and expectation of future
employee behavior, including post-vesting terminations.
We review our valuation assumptions at each grant date and, as a result, our
assumptions in future periods may change. As of September 30, 2009, total
compensation cost related to unvested stock-based awards not yet recognized was
approximately $6,200,000, which is expected to be recognized as expense over a
weighted-average period of 2.5 years. See also Note 4, "Stock-Based
Compensation," in the notes to condensed consolidated financial statements of
Part I, Item 1 of this Form 10-Q for further information.
Wind-down expenses - Rhode Island
In connection with exiting our research and manufacturing operations in
Lincoln, Rhode Island, and the relocation of our corporate headquarters and
remaining research laboratories to California in October 1999, we provided a
reserve for our estimate of the exit cost obligation. The reserve reflects
estimates of the ongoing costs of our former scientific and administrative
facility in Lincoln, which we hold on a lease that terminates on June 30, 2013.
We are seeking to sublease, assign, sell, or otherwise divest ourselves of our
interest in the facility at the earliest possible time, but we cannot determine
with certainty a fixed date by which such events will occur, if at all.
In determining the facility exit cost reserve amount, we are required to
consider our lease payments through to the end of the lease term and estimate
other relevant factors such as facility operating expenses, real estate market
conditions in Rhode Island for similar facilities, occupancy rates, and sublease
rental rates projected over the course of the leasehold. We re-evaluate the
estimate each quarter, taking account of changes, if any, in each underlying
factor. The process is inherently subjective because it involves projections
over time - from the date of the estimate through the end of the lease - and it
is not possible to determine any of the factors, except the lease payments, with
certainty over that period.
Management forms its best estimate on a quarterly basis, after considering
actual sublease activity, reports from our broker/realtor about current and
predicted real estate market conditions in Rhode Island, the likelihood of new
subleases in the foreseeable future for the specific facility and significant
changes in the actual or projected operating expenses of the property. We
discount the projected net outflow over the term of the leasehold to arrive at
the present value, and adjust the reserve to that figure. The estimated vacancy
rate for the facility is an important assumption in determining the reserve
because changes in this assumption have the greatest effect on estimated
sublease income. In addition, the vacancy rate estimate is the variable most
subject to change, while at the same time it involves the greatest judgment and
uncertainty due to the absence of highly predictive information concerning the
future of the local economy and future demand for specialized laboratory and
office space in that area. The average vacancy rate of the facility over the
last six years (2003 through 2008) was approximately 74%, varying from 66% to
89%. As of September 30, 2009, based on current information available to
management, the vacancy rate is projected to be approximately 77% for 2009,
approximately 73% for 2010 and approximately 70% from 2011 through the end of
the lease. These estimates are based on actual occupancy as of September 30,
2009, predicted lead time for acquiring new subtenants, historical vacancy rates
for the area, and assessments by our broker/realtor of future real estate market
conditions. If the assumed vacancy rate from 2010 to the end of the lease had
been 5% higher or lower at September 30, 2009, then the reserve would have
increased or decreased by approximately $146,000. Similarly, a 5% increase or
decrease in the operating expenses for the facility from 2010 on would have
increased or decreased the reserve by approximately $93,000, and a 5% increase
or decrease in the assumed average rental charge per square foot would have
increased or decreased the reserve by approximately $43,000. Management does not
wait for specific events to change its estimate, but instead uses its best
efforts to anticipate them on a quarterly basis. See Note 5 "Wind-down
expenses," in the notes to condensed consolidated financial statements of
Part I, Item 1 of this Form 10-Q for further information.
Wind-down expenses - Australia
On April 1, 2009, as part of our acquisition of the SCS operations, we
acquired operations near Melbourne, Australia. In order to reduce operating
complexity and expenses, we made the decision to close our site in Australia and
consolidate personnel and programs to our Cambridge, U.K. and Palo Alto,
California sites. U.S. GAAP requires that a liability for a cost associated with
an exit or disposal activity be recognized when the liability is incurred. In
accordance with U.S. GAAP requirements, at June 30, 2009, we established a
short-term reserve of approximately $310,000 for the estimated costs to close
down and exit our Australia operations. The reserve reflects the estimated cost
for an early termination of our facility lease in Australia (with an original
termination date of December 31, 2010), employee termination benefits and other
liabilities associated with the wind-down and relocation of our operations in
Australia.
Business Combinations
The operating results of acquired companies or operations are included in our
consolidated financial statements starting on the date of acquisition. Goodwill
is recorded at the time of an acquisition and is calculated as the difference
between the aggregate consideration paid for an acquisition and the fair value
of the net tangible and intangible assets acquired. Accounting for acquisitions
requires extensive use of accounting estimates and judgments to allocate the
purchase price to the fair value of the net tangible and intangible assets
acquired, including in-process research and development (IPR&D). Goodwill and
intangible assets deemed to have indefinite lives are not amortized but are
subject to annual impairment tests. If the assumptions and estimates used to
allocate the purchase price are not correct, or if business conditions change,
purchase price adjustments or future asset impairment charges could be required.
We test goodwill for impairment on an annual basis or more frequently if we
believe indicators of impairment exist. Impairment evaluations involve
management estimates of asset useful lives and future cash flows. Significant
management judgment is required in the forecasts of future operating results
that are used in the evaluations. It is possible, however, that the plans and
estimates used may be incorrect. If our actual results, or the plans and
estimates used in future impairment analysis, are lower than the original
estimates used to assess the recoverability of these assets, we could incur
additional impairment charges in a future period.
Results of Operations
Our results of operations have varied significantly from year to year and
quarter to quarter and may vary significantly in the future due to the
occurrence of material recurring and nonrecurring events, including without
limitation the receipt and payment of recurring and nonrecurring licensing
payments, the initiation or termination of clinical studies, research
collaborations and development programs for both cell-based therapeutic products
and research tools, unpredictable or unanticipated manufacturing and supply
costs, unanticipated capital expenditures necessary to support our business,
expenses arising out of the integration of the acquired SCS operations,
developments in on-going patent protection and litigation, the on-going expenses
to lease and maintain our Rhode Island facilities, and the increasing costs
associated with operating our California and Cambridge, U.K. facilities.
Revenue and Cost of Product Sales
Revenue for the three and nine month periods ended September 30, 2009, as
compared with the same periods in 2008, is summarized in the table below:
Three months ended, Nine months ended,
September 30 Change in 2009 versus 2008 September 30 Change in 2009 versus 2008
2009 2008 $ % 2009 2008 $ %
Revenue:
Licensing agreements
and grants $ 98,806 $ 12,379 $ 86,427 698 % $ 300,260 $ 59,561 $ 240,699 404 %
Product sales 154,362 - 154,362 * 274,961 - 274,961 *
Total revenues 253,168 12,379 240,789 1,945 % 575,221 59,561 515,660 866 %
Cost of product sales (141,453 ) - (141,453 ) * (200,979 ) - (200,979 ) *
Gross Profit $ 111,715 $ 12,379 $ 99,336 802 % $ 374,242 $ 59,561 $ 314,681 528 %
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* Calculation is not meaningful
Total revenue in the third quarter of 2009 was approximately $253,000, which
was 698% higher than total revenue in the third quarter of 2008. The increase in
2009 compared to 2008 was primarily attributable to consolidation of revenues
from the acquired SCS operations in the third quarter of 2009, which were not
part of our operations in the same period in 2008.
Third quarter ended September 30, 2009 versus third quarter ended
September 30, 2008.Licensing and grant revenue for the third quarter of 2009
were approximately $86,000, or 698%, higher as compared to the same period in
2008. This increase was primarily attributable to approximately $44,000 in grant
and licensing revenue recognized and consolidated as part of our acquisition of
the SCS operations, revenue of approximately $37,000 from an existing grant
which we were awarded in October 2008 from the National Institute of Diabetes
and Digestive and Kidney Diseases to research and develop a potential cell-based
therapeutic for liver disease, and an increase of approximately $5,000 in
licensing revenue from existing licensing agreements. In the third quarter of
2009, we
recognized approximately $154,000 and $141,000 as revenue from product sales and
cost of product sales, respectively, in connection with our acquisition of the
SCS operations, compared to none in the same period of 2008.
Nine-month period ended September 30, 2009 versus nine-month period ended
September 30, 2008.Licensing and grant revenue for the nine-month period ended
September 30, 2009 were approximately $241,000, or 404%, higher as compared to
the same period in 2008. This increase was primarily attributable to
approximately $136,000 in grant and licensing revenue recognized and
consolidated as part of our acquisition of the SCS operations, Revenue of
approximately $89,000 from an existing grant which we were awarded in
October 2008 from the National Institute of Diabetes and Digestive and Kidney
Diseases to research and develop a potential cell-based therapeutic for liver
disease, and an increase of approximately $16,000 in licensing revenue from
existing licensing agreements. For the nine months ended September 30, 2009, we
recognized and consolidated approximately $275,000 and $201,000 as revenue from
product sales and cost of product sales, respectively, in connection with of our
acquisition of the SCS operations, compared to none in the same period of 2008.
Operating Expenses
Operating expenses for the three and nine month periods ended September 30,
2009, as compared with the same periods in 2008, are summarized in the table
below:
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