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AMENDMENT NO. 1
TO
FORM 10-K
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
------------------------
/X/ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934
FOR THE FISCAL YEAR ENDED DECEMBER 31, 1999
OR
/ / TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934
COMMISSION FILE NUMBER 0-19871
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STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
(Exact name of Registrant as specified in its charter)
DELAWARE 94-3078125
(State or other jurisdiction (I.R.S. Employer Identification No.)
of incorporation or organization)
525 DEL REY AVENUE, SUITE C, SUNNYVALE, CA 94086
(Address of principal offices) (zip code)
701 GEORGE WASHINGTON HIGHWAY, LINCOLN, RI 02865
(Former address of principal executive offices) (zip code)
Registrant's telephone number, including area code: (408) 731-8670
Securities registered pursuant to Section 12(b) of the Act:
NONE
Securities registered pursuant to Section 12(g) of the Act:
COMMON STOCK, $.01 PAR VALUE
JUNIOR PREFERRED STOCK PURCHASE RIGHTS
Title of class
Indicate by check mark whether the Registrant (1) has filed all reports
required to be filed by Section 13 or 15(d) of the Securities Exchange Act of
1934 during the preceding 12 months (or for such shorter period that the
registrant was required to file such reports), and (2) has been subject to such
filing requirements for the past 90 days. Yes /X/ No / /
Indicate by check mark if disclosure of delinquent filers pursuant to Item
405 of Regulation S-K is not contained herein, and will not be contained, to the
best of registrant's knowledge, in definitive proxy or information statements
incorporated by reference in Part III of this Form 10-K or any amendment to this
Form 10-K. / /
Aggregate market value of Common Stock held by non-affiliates at March 20,
2000: $140,213,189.22. Inclusion of shares held beneficially by any person
should not be construed to indicate that such person possesses the power, direct
or indirect, to direct or cause the direction of management policies of the
registrant, or that such person is controlled by or under common control with
the Registrant. Common stock outstanding at March 20, 2000: 19,506,565 shares.
DOCUMENTS INCORPORATED BY REFERENCE
Portions of the Registrant's Definitive Proxy Statement for its 2000 Annual
Meeting of Shareholders are incorporated by reference into Part III of this
Report.
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FORWARD LOOKING STATEMENTS
This report contains certainprospectus includes forward-looking statements. You can identify these
statements regarding, among
other things, the Company's expectedby forward-looking words such as "may," "will," "possibly," "expect,"
"anticipate," "project," "believe," "estimate" and "continue" or similar words.
You should read statements that contain these words carefully because they
discuss our future expectations, contain projections of our future results of
operations or of our financial condition, or state other "forward-looking"
information. We believe that it is important to communicate our future
expectations to our investors. However, there will be events in the progress of the
Company's product developmentfuture that
we have not been able to accurately predict or control and clinical programs and of its collaborations,
the need for, and timing of, additional capital and capital expenditures,
strategic partner collaboration prospects, costs of manufacture of products, the
protection of and the need for additional intellectual property rights,
regulatory matters, the need for additional facilities and potential market
opportunities. The Company'sthat may cause our
actual results may varyto differ materially from those containeddiscussed. For example,
contaminations at our facilities, changes in suchthe pharmaceutical or biotechnology
industries, competition and changes in government regulations or general
economic or market conditions could all have significant effects on our results.
These factors should be considered carefully and readers should not place undue
reliance on our forward-looking statements. Before you invest in our common
stock, you should be aware that the occurrence of the events described in the
"Risk Factors," "Management's Discussion and Analysis of Financial Condition and
Results of Operations" and "Business" sections and elsewhere in this prospectus
could harm our business, operating results and financial condition. All
forward-looking statements because of risksattributable to whichus or persons acting on our behalf
are expressly qualified in their entirety by the Company is subject, such as risks of lack of available funding, failurecautionary statements and risk
factors contained throughout this prospectus. We are under no duty to update any
of the Companyforward-looking statements after the date of this prospectus or to
develop strategic partnerships, delays in research, adverse results
from the Company's research or development programs, obsolescence of the
Company's technology, competition from third parties, termination of the
Company's collaborations, intellectual property rights of third parties,
unavailability of needed raw materials, failure of the Company or its
collaboratorsconform these statements to perform, litigation, regulatory restrictions, and other risks
to which the Company is subject.actual results.
SEE "CAUTIONARY FACTORS RELEVANT TO FORWARD-LOOKING INFORMATION" FILED
HEREWITH AS EXHIBIT 99 AND INCORPORATED HEREIN BY REFERENCE.
2
BUSINESS
THE COMPANY
CytoTherapeutics, Inc. ("CytoTherapeutics" or the "Company") is a leaderOVERVIEW
We are engaged in research aimed at the development of noveltherapies that would
use stem cell therapies designedand progenitor cells derived from fetal or adult sources to treat, and
possibly cure, human diseases and disorders. In 1999injuries such as Parkinson's disease,
hepatitis, diabetes, and spinal cord injuries. The body uses certain key cells
known as stem cells to produce all the Company embarkedfunctional mature cell types found in
normal organs of healthy individuals. Progenitor cells are cells that have
already developed from the stem cells, but can still produce one or more types
of mature cells within an organ.
Many diseases, such as Alzheimer's, Parkinson's, and other degenerative
diseases of the brain or nervous system, involve the failure of organs that
cannot be transplanted. Other diseases, such as hepatitis and diabetes, involve
organs such as the liver or pancreas that can be transplanted, but there is a
very limited supply of those organs available for transplant. We estimate, based
on a major restructuring of its
researchinformation available to us from the Alzheimer's Association, the Centers for
Disease Control, the Family Caregiver's Alliance and development operations focusing its efforts on the discovery,
developmentSpinal Cord Injury
Information Network, that these conditions affect more than 18 million people in
the United States and commercialization of its proprietary platform stem cell
technologies, while abandoning and divesting itself of its development programs
that focused on encapsulated cell therapies. The Company's stem cellaccount for more than $150 billion annually in health care
costs.
Our proposed therapies are directed to and based on the transplantationtransplanting of healthy human stem
and progenitor cells to repair or repopulate damaged or defective neural, pancreaticreplace central nervous system, pancreas or
liver tissue that has been damaged or lost as a result of disease or injury. The Company believesinjury,
potentially returning patients to productive lives and significantly reducing
health care costs. We believe that it haswe have achieved a leadership positionsignificant progress in
research regarding stem cells of the neural stem cell therapy area with
its advancementscentral nervous system through the advances
we have made in its research and development program for the isolation, purification and transplantation of neural stem/central
nervous system stem and progenitor cells. The Company
hasWe have also made advancementsadvances in itsour
research programs to discover the stem cells of the pancreas and of the liver, and hasliver.
We have established a broadan intellectual property position with respect to stem/progenitor cell therapies in all three areas through its own patentedof our
stem cell research--the central nervous system, the pancreas and the liver--by
patenting our discoveries and entering into exclusive licensing arrangements. CytoTherapeutics, Inc. was incorporated in Delaware in 1988 and currently
has one subsidiary, StemCells California, Inc., a California corporation
acquired by the Company in September 1997.
THE UNMET NEED
Many degenerative diseases result from organ failure where organs cannot be
transplanted to cure the disease (e.g., neurodegenerative diseases and
pancreatic failure) or where there are constraints due to a short supplyWe
believe that, if successfully developed, our platform of
organs for transplant (e.g., liver). According to figures from associations for
the various diseases and government sources, these conditions, many of which
have ineffective treatments or none at all, affect more than 18 million people
in the United States and account for more than $160 billion annually in health
care costs. The Company believes its stem cell technologies
may providecreate the basis for effective therapies resulting in the replacement of certain lost or
damaged cells or regeneration of organs damaged by disease, thus potentially
returning patients to productive lives and significantly reducing health care
costs in these segments. Thus, if the Company can successfully develop and
commercialize its platform stem cell technologies, it believes that the
resulting therapies may provide the basis for addressingwould address a number of degenerative diseasesconditions
with significant unmet medical needs.
CELL THERAPY BACKGROUND
ROLE OF CELLS IN HUMAN HEALTH AND TRADITIONAL THERAPIES
In healthy individuals, cellsCells maintain normal physiological function in healthy individuals by
secreting or metabolizing substances, such as sugars, amino acids,
neurotransmitters and hormones, which are essential to life. When cells are
damaged or destroyed, they no longer produce, metabolize or accurately regulate
critical molecular substances required by the body. For example,those substances. Impaired cellular function is associated with the progressive
decline common to many neurodegenerativedegenerative diseases of the nervous system, such as
Parkinson's disease, Alzheimer's disease and amyotrophic lateral sclerosis ("ALS"), is associated
with impaired cellular function.sclerosis.
Recent advances in medical science have identified cell loss or impaired
cellular function as leading causes of degenerative diseases. Biotechnology
advances have led to the discoveryidentification of a numbersome of the specific substances or
proteins that in
certain diseasesare deficient. While administering these substances or disorders, are inadequately produced by the body's own
cells. However, while these proteins as
medication does overcome some of the limitations of traditional pharmaceuticals
such as lack of specificity, there is no existing technology that can deliver
such proteins atthem to the precise sites of action and in the appropriate physiological
quantities or for the duration required to cure the degenerative condition.
Cells, however, do this naturally. As a result, investigators have
considered using cell transplantation therapy to replace vitalreplacing failing cells that are failingno longer producing the needed
substances or proteins by implanting stem or progenitor cells that are capable of
replacing or regenerating new cells
3
to replace thosethe cell that the degenerative condition has damaged or destroyed due to the degenerative condition.destroyed.
Where there has been irreversible tissue damage or organ failure, of vital cells,
transplantation of stem cells offers the possibility of replacing the functions of these failed cells,generating new and
healthy tissue, thus potentially restoring the organ function and the patient's
health.
3
THE POTENTIAL OF OUR STEM CELL-BASED THERAPY
PLATFORM
StemWe believe that, if successfully developed, stem cell-based therapy--the use
of stem or progenitor cells to treat diseases--has the potential to provide a
broad therapeutic approach comparable in importance to traditional
pharmaceuticals and more recently, to genetically engineered biologics.
Stem cells are rare cells and are only available in limited supply, whether from the
patients themselves (autologous) or from donors
(allogeneic). Furthermore, since autologous cells aredonors. Cells obtained from the same person who will
receive them they may be abnormal if the patient is ill andor the tissue is contaminated
with disease-causing cells. Also, the cells can often can only be obtained only through
significant surgical procedures. The challenge, therefore, has been two-fold: first,three-fold:
1) to identify the stem cells and
then,cells;
2) to create techniques and processes that can be used to expand these rare
cells in sufficient quantities for effective auto-transplants, ortransplants; and
3) to establish a bank of normal human stem or progenitor cells that can be
transplantedused for transplantation into a
numberindividuals whose own cells are not
suitable because of individualsdisease or other reasons.
We have developed and which will then integrate with the endogenous cells to
repair or replace damaged cells or tissue. The Company has previously shown that
it hasdemonstrated a process, to reproducibly grow normal human brain stem/progenitor cells based on a proprietary IN
VITRO culture system in chemically defined media. The
Company believesmedia, that reproducibly grows normal
human central nervous system, or CNS, stem and progenitor cells. We believe this
is the first reproducible process for growing normal human neuralCNS stem cells. More
recently, we have discovered markers on the Company has identifiedcell surface that identify the human
neuralCNS stem cell by cell surface markers, allowing itcells. This allows us to purify them and eliminate other unwanted cell
types. Together, these discoveries enable the Companyus to purifyselect normal human neuralCNS stem
cells and to expand the numbersthem in culture to produce a large number of these cells
in culture.pure stem
cells.
Because thethese cells have not been genetically modified, they may be
especially suitable for transplantation and may provide a safer and more
effective alternative to therapies that are based on cells derived from cancer
cells, from cells modified by a cancer gene to make them grow, or from an
unpurified mixture of many different cell types. Thus, CytoTherapeutics believes
itstypes, or from animal derived cells.
We believe our proprietary stem cell technologies may provide a wayenable therapies to
replace specific cells that have been damaged or destroyed. This approach may be necessary when
cell replacement requires repair of cellular architecture or direct cell-to-cell
contact. Such replacement with stem cells may allow fordestroyed, permitting the
restoration of function through the replacement of normal cells where this has
not been possible in the past. The Company's recent advances in itsIn our research, we have shown that neuronal stem cells of
the central nervous system transplanted into hosts successfully engraft,are accepted, migrate, and
differentiatesuccessfully specialize to produce mature neurons and glial cells.
BecauseMore generally, because the stem cell is the pivotal cell in an organ that produces all
the functional mature cell types the Company believes this cell servesin an organ, we believe these cells, if
successfully identified and developed for transplantation, may serve as
a
platformplatforms for five major areas of regenerative medicine and biotechnology:
1)- tissue repair and replacement,
2)- correction of genetic disorders,
3) gene
discovery, 4)- drug discovery and screening,
- gene discovery and 5) genomics. The company isuse, and
- diagnostics.
We will be pursuing key alliances in each of these areas.
CYTOTHERAPEUTICS'4
OUR PLATFORM OF STEM CELL TECHNOLOGYTECHNOLOGIES
Stem cells may be functionally characterized ashave two defining characteristics:
- some of the cells whose progeny include
both daughterdeveloped from stem cells (by self-renewal) as well as more differentiated cells.produce all the kinds of
mature cells making up the particular organ; and
- they "self renew"--that is, other cells developed from stem cells are
themselves new stem cells, thus permitting the process to continue again
and again.
Stem cells are known to exist in humans as a self-renewing source of cells needed in the
variousfor many systems of the human body, (e.g., hematopoietic; neural, bothincluding
the blood and immune system, the central and peripheral; hepatic; pancreatic endocrine; skin;peripheral nervous systems
(including the brain), and mesenchymal stem cells).the liver, pancreas endocrine, and the skin systems.
These rare, self-renewing stem cells are present in many tissues and are responsible for organ regeneration after injury or during normal cell
replacement. The Company believesreplacement and, to a more or less limited extent, after injury. We believe that
thesefurther research and development will allow stem cells canto be cultivated and
administered in ways that enhance their natural function, so as to form the
basis of therapies that have the potential towill replace specific subsets of cells that have been
injureddamaged or lost through disease, injury or genetic defect.
The Company is seeking to identify, isolate and find methods of expanding a
variety of different human stem cells for use in treatment of a variety of human
diseases and disorders. The Company believesWe also believe that there is a finite number of
stem cells in the human system and that it is possible for the person or entity that first identifies and isolates
a given stem cell and defines methods to culture any of the finite number of different
types of human stem cells will be able to obtain patent protection for such cells.
4
The Company'sthe
methods and the composition, making the commercial development of stem cell
treatment and possible cure of currently intractable diseases financially
feasible.
Our strategy is to be the first to identify, isolate and patent multiple
types of human stem/stem and progenitor cells with commercial importance. The
Company'sOur
portfolio of issued patents includes a method of culturing normal human neural stem/central
nervous system stem and progenitor cells in itsour proprietary chemically defined
medium, and itsour published studies show that these cultured and expanded cells
give rise to all three major cell types of the central nervous system (i.e., neurons,
astrocytes, and oligodendrocytes).system. Also, a
separate study sponsored by the
Companyus using these cultured stem/stem and progenitor cells
showed that the cells are capable of transplantation into hosts, with successful engraftment, migrationaccepted, migrate, and differentiationsuccessfully specialize to
produce neurons and glial cells.
More recently, the Companywe announced the results of a new study that showed that
human braincentral nervous system stem cells can be successfully isolated by cell surface markers
present on the surface of freshly obtained brain cells. The Company believesWe believe this is the
first reproducible process for isolating highly purified populations of
well-characterized normal human neuralcentral nervous system stem cells, and hashave
applied for a composition of matter patent. Because the cells are highly
purified and have not been genetically modified, they may be especially suitable
for transplantation and may provide a safer and more effective alternative tothan
therapies that are based on cells derived from cancer cells, or from cells
modified by a cancer gene to make them grow, or from an unpurified mixture of
many different cell types. The Company hastypes or cells derived from animals. We have also filed an
improved process patent for the growth and expansion of these purified noralnormal
human neuralcentral nervous system cells.
Neurological disorders such as Parkinson's disease, epilepsy, and Alzheimer's
disease, and the side effects of stroke, affect a significant portion of the
U.S. population and there currently haveare no effective long-term therapies. The Company
believestherapies for
them. We believe that therapies based on itsour process for identifying, isolating
and culturing neural stem/stem and progenitor cells may be useful in treating such
diseases. The Company isWe are continuing itsour research into, and hashave initiated the
development of, its human neural stem/central nervous system stem and progenitor cell-based
therapies for these diseases.
The Company continuesWe continue to advance itsour research programs to discover the human
pancreatic islet stem cell
in the human pancreas and the liver stem cell. PancreaticIslet cells are the cells that
produce insulin, so islet stem cells may be useful in the treatment of Type 1
diabetes and those cases of Type 2 diabetes where insulin secretion is
defective. Liver stem cells may be useful in the treatment of diseases such as
hepatitis, cirrhosis of the liver and liver cancer.
There can be no assurance that the Company will successfully develop its
stem cell therapies. Even in the event that such therapies are successfully
developed, there can be no assurances that they will achieve the benefits
described above, that these therapies will achieve benefits therapeutically
equal to or better than the standard of treatment at time of testing, or that
the advantages of such technology will be greater than the potential
disadvantages.5
EXPECTED ADVANTAGES OF THE COMPANY'SOUR STEM CELL TECHNOLOGY
NO OTHER TREATMENT
To the best of the Company'sour knowledge, no one has developed an FDA-approved method
for replacing lost or damaged tissues from the human nervous system. Replacement
of tissues in other areas of the human body is limited to those few areassites, such
as bone marrow or peripheral blood cell transplants, where autologous transplantation of
the patient's own cells is now feasible. In a few additional areas, allogeneicincluding
the liver, transplantation of donor organs is now used, but is limited by the
scarcity of organs available through donation. The Company believesWe believe that itsour stem cell
technologies have the potential to reestablish function in at least some of the
patients who have suffered the losses referred to above.
NATURE OF REPLACEMENTREPLACED CELLS The Company believes thatPROVIDE NORMAL FUNCTION
Because stem cells can duplicate themselves, ("self-renew")or self-renew, and differentiatespecialize
into the multiple kinds of cells that are commonly lost in various diseases,
including neurodegenerative diseases. Transplantatedtransplanted stem cells may be able to migrate limited distances to the proper
location within the body, to expand and differentiatespecialize and to replace damaged or
defective cells, facilitating the return to 5
proper function. The Company believesWe believe that
such replacement of damaged or defective cells by functional cells is unlikely
to be achieved with any other treatment.
RESEARCH EFFORTS AND PRODUCT DEVELOPMENT PROGRAMS
OVERVIEW OF RESEARCH AND PRODUCT DEVELOPMENT STRATEGY
The Company hasWe have devoted substantial resources to itsour research programs to isolate
and develop a series of stem/stem and progenitor cells that the Company believeswe believe can serve as a
basis for replacing diseased or injured cells. Stem cells are
rare, undifferentiated cells that can both self-renew and produce differentiated
(functionally specialized) cell types that constitute the various tissues or
organ system of the human body. The focus of the Company'sOur efforts to date have been
directed at methods to identify, isolate and culture a varietylarge varieties of stem/stem and
progenitor cells of the human nervous system, liver and pancreas and to develop
therapies utilizing these stem/stem and progenitor cells.
The following table lists the potential therapeutic indications for, and
current status of, CytoTherapeutics'our primary research and product development programs and
projects andprojects. The table is qualified in its entirety by reference to the more
detailed descriptions of such programs and projects appearing elsewhere in this
Report. The Companyprospectus. We continually evaluates itsevaluate our research and product development efforts
and reallocatesreallocate resources among existing programs or to new programs in light of
experimental results, commercial potential, availability of third party funding,
likelihood of near-term efficacy, collaboration success or significant
technology
6
enhancement, as well as other factors. The Company'sOur research and product development
programs are at relatively early stages of development and will require
substantial resources to commercialize.
There can be no assurance
that the Company will successfully develop any product or obtain regulatory
approvals, enter clinical trials, achieve other milestones or commercialize any
products in accordance with currently anticipated timetables, or at all.
RESEARCH AND PRODUCT DEVELOPMENT PROGRAMS
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PROGRAM DESCRIPTION AND OBJECTIVE STAGE/STATUS(1)
- --------------------------------- ------------------------------------------------------------
HUMAN NEURAL STEM CELL PRECLINICAL
Repair or replace damaged CNScentral - Demonstrated IN VITRO the ability to initiate and expand
stemnervous system tissue (including retinalstem cell-containing human neural cultures and
differentiation
degeneration and the resultsspinal cord, degenerated retinas specialization into three types of CNScentral nervous system
and tissue affected by certain cells
of certain
genetic disorders) - Direct isolationDemonstrated the ability of neurosphere-initiating stem
cells from human brain
- IN VIVO demonstration of proper differentiation and
engraftment ofDemonstrated in rodent studies that transplanted human
neural cell cultures containing CNSbrain-derived stem cells in rodent CNS
PANCREATICare accepted and properly
specialized into the three major cell types of the
central nervous system.
PANCREAS ISLET STEM CELL RESEARCH
Repair or replace damaged pancreas - Identified cellmarkers on the surface marker usedof cells to identify,
isolate
pancreatic islet tissue isolate and culture pancreatic islet stem cells of the pancreas
- CommencingCommenced small animal testing
LIVER STEM CELL RESEARCH
Repair or replace damaged Discovery program to identify, isolate and patent humanliver - Demonstrated the production of hepatocytes from purified
tissue including tissue resulting mouse hematopoietic stem liver tissue (including the cells
for the liver
results offrom certain metabolic genetic diseases)- Identified IN VITRO culture assay for growth of human
diseases bipotent liver progenitor cells that can produce both
bile duct and hepatocytes
- Showed that the in vitro culture of human bipotent liver
cells can also grow human hepatitis virus
- ------------------------
(1) "Research" refers to early stage research and product development activities
IN VITRO, including the selection and characterization of product candidates
for preclinical testing. "Preclinical" refers to further testing of a
defined product candidate IN VITRO and in animals prior to clinical studies.
6
RESEARCH AND DEVELOPMENT PROGRAMS
The Company'sOur portfolio of stem cell technology results from the Company'sour exclusive licensing
of neural stem/central nervous system, stem and progenitor cell technology, animal models
for the identification and/or testing of stem and other
technologies applicable to the pancreasprogenitor cells and liver, the Company'sour own
research and development efforts to date, and the acquisition of StemCells, Inc. (now
renamed StemCells California, Inc.) in 1997. The Company, through its
subsidiary, StemCells California, Inc., has been advancing its program directed
to the discovery, isolation and culturing of various stem cells from the human
body. The Company believesdate. We believe that therapies using stem
cells represent a fundamentally new approach to the treatment of diseases caused
by lost or damaged tissue. The Company hasWe have assembled an experienced team of scientists
and scientific advisors to consult with and advise the Company'sour scientists on their
continuing research and development of stem/stem and progenitor cells. This team
includes, among others, Irving L. Weissman, M.D., of Stanford University, Fred
H. Gage, Ph.D., of The Salk Institute and David Anderson, Ph.D., of the
California Institute of Technology.
NEURAL STEM/BRAIN STEM AND PROGENITOR CELL RESEARCH AND DEVELOPMENT PROGRAM
The CompanyWe began itsour work with neural stem/central nervous system stem and progenitor cell
cultures in collaboration with NeuroSpheres, Ltd., in 1992. The Company believesWe believe that
NeuroSpheres was the first to invent these cultures and NeuroSpheres has filed
patent applications on its inventions relating to these cultures. The Company isWe are the exclusive,
worldwide licensee from NeuroSpheres to such inventions and associated patents
and patent applications for all uses, including transplantation in the human
body as embodied in these patents. See "License Agreements and Sponsored
Research Agreements -NeuroSpheres,Agreements--NeuroSpheres, Ltd."
In December 1998, the Company announced that the US Patent and Trademark Office had
granted patent No. 5,851,832, covering the Company's methods for the human
neural cell cultures containing central nervous system stem cells, for
compositions of human neural cell cultures expanded by these methods, and for
use of these cultures in, e.g., human transplantation and remyelination. These
human neural stem/progenitor cell cultures may be useful for repairing or
replacing damaged central nervous system tissue, including the brain and the
spinal cord.
Previously, in 1997, Companyour scientists had invented a reproducible method for isolating and growing human
neural stem/CNS, stem and progenitor cells in cultures. In preclinical IN-VITROIN VITRO and early IN-VIVOIN
VIVO studies, the Companywe demonstrated that these cells differentiatespecialize
7
into all three of the cell types of the CNS. Based oncentral nervous system. Because of these
results, the Company believeswe believe that these cells may form the basis for replacement of cells
lost in certain degenerative diseases. The Company isWe are continuing research into, and hashave
initiated the development of, itsour human neural
stem/CNS stem and progenitor cell cultures.
The Company hasWe have initiated the cultures and demonstrated that these cultures can be
expanded for a number of generations IN VITRO in chemically defined media. In
collaboration with the Company,us, Dr. Anders Bjorklund has shown that cells from these
cultures can be successfully engraftedtransplanted and accepted into the brains of
rodents where they subsequently migrated and differentiatedspecialized into the appropriate
cell lineagestypes for the site of the brain into which they were transplanted.placed.
In 1998, the Companywe expanded itsour preclinical efforts in this area by initiating
programs aimed at the discovery and use of specific monoclonal antibodies to
facilitate identification and isolation of neuralCNS and other stem and progenitor
cells or their differentiatedspecialized progeny. Also in 1998, Companyour researchers devised
methods to advance the IN-VITROIN VITRO culture and passage of human neuralCNS stem cells that have
resulted in a 100-fold increase in CNS stem and progenitor cell production
of these neural stem/progenitor cells after
6 passages. The Company isWe are expanding itsour preclinical efforts toward the goal of
selecting the proper indications to pursue.
In December 1998, we announced that the US Patent and Trademark Office had
granted patent No. 5,851,832, covering our methods for the human CNS cell
cultures containing central nervous system stem cells, for compositions of human
CNS cells expanded by these methods, and for use of these cultures in human
transplantation. These human CNS stem and progenitor cells expanded in culture
may be useful for repairing or replacing damaged central nervous system tissue,
including the brain and the spinal cord.
In October 1999, the US Patent and Trademark Office granted patent number
5,968,829 entitled "Human CNS Neural Stem Cells," covering the Company'sour composition of
matter patent for human CNS neural stem cells, and also allowed a separate patent
application for itsour media for culturing human CNS neural stem cells.
Also in 1999, the Companywe announced the filing of a US patent application covering
itsour proprietary process for the direct isolation of normal human neuralCNS stem cells
based on the cell surface markers found to be present on the surface of freshly obtained
brain cells. Since the filing of this patent application, Companyour researchers have
completed a study designed to identify, isolate and culture human neuralCNS stem
7
cells
utilizing this proprietary process. In November 1999, the Companywe announced the study's
first results: Our researchers, by using itsour proprietary cellmarkers on the surface
markers,
Company researchersof the cell, had succeeded in identifying, isolating and purifying human neuralCNS
stem cells from brain tissue, and have expandedwere able to expand the number of these cells
in culture.
The Company believesWe believe that this is the first study to show a reproducible process for
isolating highly purified populations of well-characterized normal human neuralCNS
stem cells. Because the cells are normal human neuralCNS stem cells and have not been
genetically modified, they may be especially suitable for transplantation and
may provide a safer and more effective alternative to therapies that are based
on cells derived from cancer cells or from an unpurified mix of many different
cell types.types, or from animal derived cells.
In January 2000, the Companywe reported what it regardswe regard as an even more important
result: that, inIn long term animal studies, itsour researchers were able to take these
purified and expanded stem cells and transplant them into intactnormal mouse brain
hosts, where they engrafttake hold and grow into neuronalneurons and glial cells.
During the course of the study, the transplanted human neuralCNS stem cells
survived for as long as seven monthsone year and had migrated to specific functional domains of
the host brain, with no sign of tumor formation or adverse effects on the animal
recipients; moreover, the cells were still dividing. These findings show that
the neuralwhen CNS stem cells isolated and cultured utilizing the Company'swith our proprietary processes whenare
transplanted, they adopt the characteristics of the host brain and act like
normal stem cells, and suggestcells. In other words, the study suggests the possibility of a
continual replenishment of normal human neuralbrain cells.
Human neural stem/As noted above, human CNS stem and progenitor cells harvested and purified
and expanded using the Company'sour proprietary processes may be useful for creating
therapies for the treatment of neurodegenerativedegenerative brain diseases such as Parkinson's,
Huntington's and Alzheimer's disease, and other neurologicaldisease. These conditions that affect the central nervous
system, such as stroke and epilepsy--diseases and conditions that affect more than
8
5 million people in the United States and for whichthere are no effective long-term
therapies are currently available. The Company believes thatWe believe the ability to isolatepurify human brain stem
cells directly from fresh uncultured tissue is important for other reasons as well.
First,because:
- it provides aan enriched source of genetically unmodified, normal stem cells, for
transplantation, uncontaminatednot contaminated by
other unwanted or diseased cell types.
Second,types, that can be expanded in culture
without fear of also expanding some unwanted cell types;
- it opens the way to a better understanding of the properties of these
cells and how they might be manipulated in order to treat specific diseases. For
example, in certain genetic diseases such as Tay Sachs and Gaucher's, a
key metabolic enzyme required for normal development and function of the
brain is absent. Brain-derived stem cell-derived neuralcell cultures canmight be genetically
modified to secrete needed proteins for the brain. Finally,produce those proteins. The modified brain stem cells could be
transplanted into patients with these genetic diseases;
- the efficient engraftmentacceptance of these non-transformed normal human stem cells
into host brains means that the cell product can be tested in animal
models for its ability to correct deficiencies caused by various human
neurological diseases. This technology could also provide a unique animal
model for the testing of drugs that act on human brain cells either for
effectiveness of the drug against the disease or its toxicity to human
nerve cells.
The Company's neural stem/progenitor cell program is at an early stage and
there can be no assurance that it will result in any commercial product.
PANCREATIC AND LIVERPANCREAS STEM CELLS DISCOVERY RESEARCH PROGRAMS
The Company'sOur discovery program directed to the identification, isolation and
culturing of the pancreatic stem/pancreas stem and progenitor cellcells is currently being conducted
by Nora Sarvetnick, Ph.D., of The Scripps Research Institute, in collaboration
with some of our senior researchers from the Company.researchers.
According to diabetes and juvenile diabetes foundations, between 800,000 and
1.5 million Americans have Type 1 diabetes, (oftenwhich is often called "juvenile
diabetes" and most commonly diagnosed in childhood);childhood; and 30,000 new patients are
diagnosed with the disease every year. It is a costly, serious, lifelong
condition, requiring constant attention and insulin injections every day for
survival.
About 15 million other people in the United States have Type 2 diabetes
mellitus, which is also a chronic and potentially fatal condition; and more than
700,000 new patients are diagnosed annually.
Diabetes is widely recognized as one of the leading causes
of death and disability in the United States and is associated with long term
complications that affect almost every major part of the body. Diabetes-related
treatment costs exceed $100 billion annually. In 1998, the Companywe obtained an exclusive, worldwide license from The Scripps
Research Institute to novel technology developed by Dr. Sarvetnick as a result of the research sponsored by
the Company, which may
facilitate the identification and isolation of pancreatic
stem/pancreas stem and progenitor
cells by using a mouse model that continuously regenerates the pancreas. We
believe that stem cells produce the regeneration, in which case this animal
model may be useful for identifying specific markers on the cell surface markers unique
to 8
thesethe pancreas stem cells. The Company believesWe believe this may lead to the development of
cell-based treatments for Type 1 diabetes and that portion of Type 2 diabetes
characterized by defective secretion of insulin.
In 1999, advances in the research sponsored by the Companyus resulted in the Company'sour obtaining
additional exclusive, worldwide licenses from The Scripps Research Institute to
novel markers on the cell surface
markers identified by Dr. Sarvetnick and her research
team as being unique to the pancreaticpancreas islet stem cell for which the Company haswe have now filed
a US patent application. Company researchers, inIn collaboration with Dr. Sarvetnick, we continue to
advance theirthe discovery program directed toat the identification, isolation and
culturing of the pancreatic stem/pancreas stem and progenitor cellcells utilizing this technology.
The CompanyLIVER STEM CELLS DISCOVERY RESEARCH PROGRAMS
We initiated itsour discovery work for the liver stem/stem and progenitor cell
through a sponsored research agreement with Markus Grompe, Ph.D., of Oregon
Health Sciences University. Dr. Grompe's work focuses on the discovery and
development of a suitable method for identifying and assessing liver stem/stem and
progenitor cells for use in transplantation. We have also obtained a worldwide
exclusive license to a novel mouse model of liver failure for evaluating cell
transplantation developed by Dr. Grompe.
9
Approximately 1 in 10 Americans suffers from diseases and disorders of the
liver for which there are currently no effective, long-term treatments.
In 1998, Companyour researchers continued to advance methods for establishing
enriched cell populations suitable for transplantation in preclinical animal
models for evaluating these methods.
The Company ismodels. We are focused on discovering and utilizing itsour proprietary methods to
identify, isolate and culture liver stem/stem and progenitor cells and to evaluate
these cells in preclinical animal models.
In 1999, theour researchers devised a culture assay for facilitating the identification of athat we will use in our
efforts to identify liver stem/stem and progenitor cell.cells. In addition to supporting
the growth of an early human liver stem/bipotent progenitor cell, it is also possible
to infect this culture with human hepatitis virus, so it
providesproviding a valuable system
for study of the virus. This technology could also provide a unique animalIN VITRO
model for the testing of drugs that act on, or are metabolized by, human liver
cells.
An important element of the Company'sour stem cell discovery program is the further
development of intellectual property positions with respect to stem and
progenitor cells. The Company hasWe have also obtained rights to certain inventions relating to
stem cells from, and isare conducting stem cell related research at, several
academic institutions. See "License Agreement and Sponsored Research
Agreements." The Company expectsWe expect to expand itsour search for new stem/stem and
progenitor cells and to seek to acquire rights to additional inventions relating
to stem/stem and progenitor cells from third parties.
The Company's pancreatic and liver stem/progenitor cells programs are at an
early stage and there can be no assurance that they will result in any
commercial products.
WIND-DOWN OF ENCAPSULATED CELL TECHNOLOGYTHERAPY RESEARCH AND DEVELOPMENT PROGRAMS
Until mid-1999, CytoTherapeuticswe engaged in research and development in encapsulated cell
therapy technology, ("ECT"),or ECT, including a pain control program funded by Astra, and later
AstraZeneca Group plc. The results from the 85-patient double-blind,
placebo-controlled trial of CytoTherapeutics'our encapsulated bovine cell implant for the
treatment of severe, chronic pain in cancer patients did not, however, meet the
criteria AstraZeneca had established for continuing trials for the therapy. Intherapy, and
in June 1999, AstraZeneca terminated the collaboration.
Consequently, in July 1999, the Companywe announced plans for the restructuring of itsour
research operations to abandon all further ECT research and to concentrate itsour
resources on the research and development of itsour proprietary platform of stem
cell technology platform. The Companytechnology. We reduced itsour workforce by approximately 68 full-time
employees who had been focused on ECT programs, wound down itsour research and
manufacturing operations in Lincoln, Rhode Island, and relocated itsour remaining
research and development activities, and itsour corporate headquarters, to the
facilities of itsour wholly owned subsidiary, StemCells California, Inc., in
Sunnyvale, California. The Company isWe are actively marketing
the quarters it had occupied in Rhode Island, seeking to sublease, assign or sell itsour
interest in itsour former corporate headquarters building and itsour pilot
manufacturing and cell processing facility there. (SEE ALSO "LIQUIDITY AND
CAPITAL RESOURCES" UNDER MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL
CONDITION AND RESULTS OF OPERATIONS.)
9
in Rhode Island.
In December 1999 the Companywe sold itsour intellectual property assets related to itsour ECT
to Neurotech S.A., a privately held French company, in exchange for a payment of
$3 million, royalties on future product sales, and a portion of certain revenues
Neurotech may in the future receive from third parties. The
CompanyWe retained certain
non-exclusive rights to use the ECT in combination with itsour proprietary stem
cell technology, and in the field of vaccines for prevention and treatment of
infectious diseases.
(SEE ALSO "LIQUIDITY AND
CAPITAL RESOURCES" UNDER MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL
CONDITION AND RESULTS OF OPERATIONS AND NOTE 2--"PATENT COSTS" TO THE
ACCOMPANYING FINANCIAL STATEMENTS.)
In a related development, by mutual consent the Companywe and the Advanced Technology
Program of the National Institute of Standards and Technology terminated two
grants previously awarded to the Companyus for itsour encapsulated cell therapy and stem
cell-related research. The encapsulated cell therapy grant was obviated by the
sale of the technology to Neurotech. The funding agency has invited CytoTherapeuticsus to
resubmit a proposal consistent with the new directions the Company iswe are taking in itsour
research and development of itsour platform of stem cell technology.technologies.
10
SUBSIDIARY
STEMCELLS CALIFORNIA, INC.
On September 26, 1997, CytoTherapeuticswe acquired by merger StemCells, Inc. (now StemCells
California, Inc.), a California corporation, ("StemCells").
CytoTherapeutics acquired StemCells in exchange for 1,320,691 shares of
the
Company'sour common stock and options and warrants for the purchase of 259,296 common
shares. Simultaneously with the acquisition, its President, Richard M. Rose,
M.D., President of StemCells, became our President, Chief Executive Officer and a director,
of CytoTherapeutics, and Irving
L. Weissman, M.D., a founder of StemCells,the California corporation, became a directormember of
CytoTherapeutics. The Company,our board of directors. We, as the sole stockholder of StemCells,our subsidiary, voted on
February 23, 2000, to amend theits Certificate of Incorporation
of StemCells, Inc., in order to change its name
to StemCells California, Inc.
CORPORATE INVESTMENT
In July 1996, we, together with certain founding scientists, established
Modex Therapeutics SA, a Swiss biotherapeutics company, to pursue extensions of
our former technology of ECT for certain applications outside the central
nervous system. Modex, headquartered in Lausanne, Switzerland, was formed to
integrate technologies developed by us and by several other institutions to
develop products to treat diseases such as diabetes, obesity and anemia. After
our disposition of the encapsulated cell technology in December 1999, we no
longer had common research or development interests with Modex, but we held
approximate 17% of its stock. Modex completed an initial public offering on
June 23, 2000, in the course of which we realized a gain of approximately
$1.4 million from the sale of certain shares. We now own 126,193 shares, or
approximately 9%, of Modex's equity, subject to a lockup until December 23,
2000. The Company's current stem cellclosing market price of Modex stock on the Swiss Neue Market exchange
on October 31, 2000, was 329.5 Swiss Francs, or approximately $183, per share.
LICENSE AGREEMENTS AND SPONSORED RESEARCH AGREEMENTS
We have entered into a number of license agreements with commercial and
non-profit institutions, as well as a number of research-plus-license agreements
with academic organizations. The research is being conducted pursuantagreements provide that we will fund
certain research costs, and in return, will have a license or an option for a
license to the provisions of an agreement between CytoTherapeutics and Drs. Weissman and Gage
providing for a two-year research plan. Ifresulting inventions. Under the goals of the research plan are
accomplished, the stem cell researchlicense agreements, we will
continue totypically be funded under an
extension of such Research Plan approved by a Research Committee consisting of
two persons chosen by Drs. Weissman and Gage, two persons chosen by the Company
and a fifth member appointed by Drs. Weissman and Gage, subject to obligations of due diligence and the reasonable approval of the Company. Increases in stem cell research funding of
not more than 25% a year approved by the Committee will be funded by
CytoTherapeutics (although CytoTherapeutics also retains the rightrequirement to fundpay
royalties on products that use patented technology licensed under such
programs at a higher level) for as long as the goals of the Research Plan are
being met, provided, however, that CytoTherapeutics will retain the option of
(i) ceasing or reducing neural stem research even if all Research Plan goals are
being met by accelerating the vesting of all still-achievable performance-based
options granted to Drs. Weissman and Gage and other scientists and (ii) ceasing
or reducing non-neural stem cell research even if all Research Plan goals are
being met by affording StemCells' scientific founders the opportunity to
continue development of the non-neural stem research by licensing the technology
related to such research to them in exchange for a payment to CytoTherapeutics
equal to all funding for such research, plus royalty payments.
CORPORATE COLLABORATIONSagreements.
ASTRAZENECA PLC
In March 1995, CytoTherapeuticswe signed a collaborative research and development agreement
with Astra (later AstraZeneca plc)plc for the development and marketing of certain
encapsulated-cell products to treat pain. Under the agreement, the Companywe conducted
research and development and received approximately $42 million, including
research and development funding, through June 1999 when, as noted above (SEE
WIND-DOWN OF ENCAPSULATED CELL THERAPYTECHNOLOGY RESEARCH AND DEVELOPMENT PROGRAMS)PROGRAM),
AstraAstraZeneca exercised its right to terminate the agreement. (SEE ALSO LIQUIDITY
AND CAPITAL RESOURCES UNDER MANAGEMENT'S 10
DISCUSSION AND ANALYSIS OF FINANCIAL
CONDITION AND RESULTS OF OPERATIONS AND NOTE 15--17--"RESEARCH AGREEMENTS" TO THE
ACCOMPANYING FINANCIAL STATEMENTS.)
MODEX THERAPEUTIQUES SAGENENTECH, INC.
In JulyNovember 1996, CytoTherapeutics, togetherwe signed collaborative development and licensing
agreements with Genentech relating to the development of products using the
Company's ECT technology to deliver certain founding scientists,
established Modex Therapeutiques SA ("Modex"), a Swiss biotherapeutics company,
to pursue extensions of CytoTherapeutics' broad-based, encapsulated-cell
technology for certain applications outside the central nervous system. Modex,
headquartered in Lausanne, Switzerland, was formed to integrate technologies
developed at three universities in Lausanne (the University of Lausanne, the
Centre Hospitalier Universitaire Vaudois, and the Ecole Polytechnique Federale
de Lausanne), at the Albert Einstein College of Medicine of Yeshiva University
in New York City, and at CytoTherapeutics, to develop productsGenentech's proprietary growth
factors to treat non-CNS
diseases such as diabetes, obesityParkinson's disease, Huntington's disease and anemia. In October 1997,amyotrophic
lateral sclerosis.
Under the Company
completed a series of transactions that resulted in the establishment of Modex
as an independent company in which CytoTherapeutics has an equity position of
approximately 17%. The pre-existing Cross License Agreement between the Company
and Modex, which concerned encapsulated cell technology, was assigned to
Neurotech, S.A. in December 1999 as partterms of the divestitureagreement for Parkinson's disease, Genentech had the
right, at its discretion, to terminate the program at specified milestones. On
May 21, 1998, Genentech exercised its right to
11
terminate the Parkinson's collaboration. Pursuant to the terms of the agreement,
Genentech demanded that we redeem certain shares of the Company's ECT.redeemable
common stock held by them for approximately $3.1 million, at a price of $10.01
per share. This amount, per the agreement, was equal to the funds invested by
Genentech to acquire such stock less the amount we expended on the terminated
program. In March 2000, we announced a settlement of this claim at no cost to
us, and also terminated the Huntington's disease and ALS agreements. (FOR
FURTHER DETAILS AND INFORMATION REGARDING THIS SETTLEMENT, SEE LIQUIDITY AND
CAPITAL RESOURCES UNDER MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL
CONDITION AND RESULTS OF OPERATIONS.)
STATE OF RHODE ISLAND
In 1989 we entered into an agreement with the Rhode Island Partnership for
Science and Technology, or RIPSAT, for reimbursement of $1,172,000 for certain
research activities we funded at Brown University. Under the terms of this grant
we were obligated to pay royalties ranging from three to five percent of
revenues from products developed under the agreement, to a maximum of
$1,758,000. In July 1999, when we announced our plans to terminate ECT research,
wind down operations in Rhode Island and relocate research activities and
corporate headquarters to Sunnyvale, California, RIPSAT alleged that we were in
default under this funding agreement. While we believed that we were not in
default, in March 2000, we entered into a settlement of the claim. (FOR FURTHER
DETAILS AND INFORMATION REGARDING THIS SETTLEMENT, SEE LIQUIDITY AND CAPITAL
RESOURCES UNDER MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND
RESULTS OF OPERATIONS.)
NEUROSPHERES, LTD.
In March 1994, we entered into a Contract Research and License Agreement
with NeuroSpheres, Ltd., which was clarified in a License Agreement dated as of
April 1, 1997. Under the agreement as clarified, we obtained an exclusive patent
license from NeuroSpheres in the field of transplantation, subject to a limited
right of NeuroSpheres to purchase a nonexclusive license from us, which right
was not exercised and has expired. We have developed additional intellectual
property relating to the subject matter of the license. We entered into an
additional license agreement with NeuroSpheres as of October 31, 2000, under
which we obtained an exclusive license in the field of non-transplant uses, such
as drug discovery and drug testing, so that together the licenses are exclusive
for all uses of the technology. We will make up-front payments to NeuroSpheres
of 65,000 shares of our common stock and $50,000, and additional cash payments
when milestones are achieved in the non-transplant field, or in any products
employing NeuroSpheres patents for generating cells of the blood and immune
system from neural stem cells. Milestone payments would total $500,000 for each
product that is approved for market. Our agreements with NeuroSpheres will
terminate at the expiration of all patents licensed to us, but can terminate
earlier if we breach without curing our obligations under the agreement or if we
declare bankruptcy. We would have a security interest in the licensed technology
in the event that NeuroSpheres declares bankruptcy.
SIGNAL PHARMACEUTICALS, INC.
In December 1997, StemCells California, Inc.we entered into two license agreements with Signal
Pharmaceuticals, Inc. under which each party licensed to the other certain
patent rights and biological materials for use in defined fields. An initial
disagreement as to the interpretation of the rights licensed to StemCells California, Inc.rights was resolved by the
parties, and the agreements are operating in accordance with their terms. GENENTECH, INC.
In November 1996,Signal
has now been acquired by Celgene. Each agreement with Signal will terminate at
the Company signed collaborative development andexpiration of all patents licensed under it, but the licensing agreements with Genentech relatingparty can
terminate earlier if the other party breaches its obligations under the
agreement or declares bankruptcy. Also, the party receiving the license can
terminate the agreement at any time upon notice to the development of products using the
Company's encapsulated cell therapy technology to deliver certain of Genentech's
proprietary growth factors to treat Parkinson's disease, Huntington's disease
and amyotrophic lateral sclerosis ("ALS").other party. Under the terms of the agreementthese
agreements, we must reimburse Signal for Parkinson's disease, Genentech had the
right, in its discretion, to terminate the Parkinson's program at specified
milestones in the program, and on May 21, 1998, Genentech exercised its right to
terminate the Parkinson's collaboration. Pursuantpayments it must make to the termsUniversity
of the
Parkinson's Agreement, upon termination Genentech demanded that the Company
redeem, at a priceCalifornia based on products we develop and for 50% of $10.01 per share, certain shares of the Company's
redeemable Common Stock held by Genentech, in an amount equal to the amount of
funds invested by Genentech to acquire such stock less the amount expended by
the Company on the terminated program, a difference of approximately
$3.1 million. In March 2000, the Company announced a settlement of this claim at
no cost to the Company. The Huntington's disease and ALS agreements have been
terminated as well. (FOR FURTHER DETAILS AND INFORMATION REGARDING THIS
SETTLEMENT, SEE LIQUIDITY AND CAPITAL RESOURCES UNDER MANAGEMENT'S DISCUSSION
AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS.)
LICENSE AGREEMENTS ANDother payments
Signal must make.
12
SPONSORED RESEARCH AGREEMENTS
NEUROSPHERES, LTD.
In March 1994, the Company entered into a Contract Research and License
Agreement with NeuroSpheres, Ltd. Under the agreement, the Company obtained from
NeuroSpheres an exclusive, worldwide, royalty-bearing license for the commercial
development and use of certain neural stem cells for transplantationWhen we decided to treat
human disease. In 1997, the Company settled a dispute that arose between it and
NeuroSpheres under the agreement. Pursuant to the settlement, the Company
obtained an exclusive
11
patent license from NeuroSpheres in the field of transplantation, subject to a
limited right of NeuroSpheres to purchase a nonexclusive license from the
Company. Such right was not exercised by Neurospheres and expired in
April 1998. The Company and NeuroSpheres have no further research obligations to
one another. The Company has developed additional intellectual property relating
to the subject matter of the license.
STATE OF RHODE ISLAND
In 1989, the Company entered into an agreement with the State of Rhode
Island pursuant to which the Rhode Island Partnership for Science and Technology
("RIPSAT"), an agency of the State, reimbursed the Company $1,172,000 for
certain research activities the Company funded at Brown University. Under the
terms of this grant, the Company is obligated to pay royalties ranging from
three to five percent of revenues from products developed under the agreement,
to a maximum of $1,758,000. In July 1999, when the Company announced its plans
to proceed no further with its ECT research, wind down its operations in Rhode
Island and relocate its research activities and corporate headquarters to
Sunnyvale, California, RIPSAT alleged that the Company was in default under this
funding agreement. While the Company believed that it was not in default, in
March 2000, the Company entered into a settlement of the claim. (FOR FURTHER
DETAILS AND INFORMATION REGARDING THIS SETTLEMENT, SEE LIQUIDITY AND CAPITAL
RESOURCES UNDER MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND
RESULTS OF OPERATIONS.)
ACADEMIC RELATIONSHIPS
The Company and its wholly owned subsidiary, StemCells California, Inc.,
have entered into a number of research and/or license agreements with academic
organizations. These research agreements provide that the Company will fund
certain research costs and, in return, will have a license or an option for a
license to the resulting inventions. Under these license agreements, the Company
and/or StemCells California, Inc. will typically be subject to obligations of
due diligence and the requirement to pay royalties on products that use patented
technology licensed under such agreements.
CYTOTHERAPEUTICS, INC.
The Company has historically expended substantial sums to support academic
research programs. However, with the decision by the Company to abandon its further research and development of its encapsulated cell therapyour ECT
technology the
Companyin July 1999, we terminated itsour academic collaborations with Brown
University and Dr. Patrick Aebischer at the Centre Hospitalier Universitaire
Vaudois in Switzerland. Research and development expenses paid in connection
with these collaborations aggregated approximately $156,600, $701,000 and
$1,326,000 for the years ended December 31, 1999, 1998, and 1997, respectively.
The Company
also has a number of collaborative relationships with other academic
institutions and academic researchers.
STEMCELLS CALIFORNIA, INC.
StemCells California, Inc. has entered into a number of research agreements
with academic institutions. Under Sponsored Research Agreements with The Scripps Research Institute and
Oregon Health Sciences University, StemCells
California, Inc.we funded certain research (in the amounts of approximately
$77,000 and $28,000 in 1997, $307,000 and $251,000 in 1998, and $309,000 and
$172,000 in 1999, for Scripps and Oregon respectively) in return for
licenses or options to license the inventions resulting from suchthe research. StemCells
California, Inc. hasWe
have also entered into license agreements with the California Institute of
Technology. All of these agreements relate largely to stem or progenitor cells
and or to processes and methods for the isolation, identification, expansion or
culturing of stem or progenitor cells. 12
We paid Scripps and Oregon respectively
approximately $77,000 and $28,000 in 1997, $307,000 and $251,000 in 1998, and
$309,000 and $172,000 in 1999 under these agreements.
Our research agreement with Scripps expires on November 14, 2000 and we are
negotiating with Scripps to extend the term of this agreement or to enter into a
new agreement. As of the date of this report, we have not yet completed our
negotiations with Scripps and we cannot give any assurance that our negotiations
will be successful. If we are unable to extend the term of this agreement, we
will have to find a replacement to perform this research or we will have to
perform this research ourselves. In either case, we may experience delay and
additional expense in connection with this research effort. Our license
agreements with Scripps will terminate upon expiration, revocation or
invalidation of the patents licensed to us, unless governmental regulations
require a shorter term. These license agreements also will terminate earlier if
we breach without curing our obligations under the agreement or if we declare
bankruptcy, and we can terminate the license agreements at any time upon notice.
Upon the initiation of the Phase II trial for our first product using Scripps
licensed technology, we must pay Scripps $50,000 and upon completion of that
Phase II trial we must pay Scripps an additional $125,000. Upon approval of the
first product for sale in the market, we must pay Scripps $250,000.
Our license agreements with the California Institute of Technology will
expire upon expiration, revocation, invalidation or abandonment of the patents
licensed to us. We can terminate any of these license agreements by giving 30
days' notice to the California Institute of Technology. Either party can
terminate these license agreements upon a material breach by the other party. We
paid $10,000 to the California Institute of Technology upon execution of the
license agreements, and we must pay an additional $10,000 upon the issuance of
the patent licensed to us under the relevant agreement. We also will pay $5,000
on the anniversary of the issuance of the patent licensed to us under the
relevant agreement. These amounts are creditable against royalties we must pay
under the license agreements. The maximum royalties that we will have to pay to
the California Institute of Technology will be $2 million per year, with an
overall maximum of $15 million. Once we pay the $15 million maximum royalty, the
licenses will become fully paid and irrevocable.
MANUFACTURING
The keys to successful commercialization of neural stem/brain stem and progenitor cells
are expected to include efficacy, safety, and consistency of the product, and economy of the process. The Company expectsWe
expect to address these issues by appropriate testing and banking representative
vials of large-scale cultures. Commercial production is expected to involve
expansion of banked cells and packaging them in an appropriate container(s)containers after
formulating the cells in an effective carrier. The carrier (which may also be used to
affectimprove the stability and engraftingacceptance of the stem cells or their progeny).progeny. Because
of the early stage of the Company's
stem/our stem and progenitor cell programs, all of the issues
that will affect manufacture of stem/stem and progenitor cell products are relatively unclear at this juncture.not yet
clear.
13
MARKETING
The Company expectsWe expect to market and sell itsour products primarily through co-marketing,
licensing or other arrangements with third parties. The Company
does not have experience in sales,There are a number of
substantial companies with existing distribution channels and large marketing
or distributionresources who are well equipped to market and does not expect
to develop such capabilities in the near future. Generally, the Company intendssell our products. It is our
intent to have the marketing of itsour products undertaken by such partners,
although the
Companywe may seek to retain limited marketing rights in specific narrow
markets
particularly where the product may be addressed by a specialty or niche sales force.
PATENTS, PROPRIETARY RIGHTS AND LICENSES
The Company believesWe believe that proprietary protection of itsour inventions will be of major
importance to itsour future business. The Company has aWe have an aggressive program of vigorously
seeking and protecting our intellectual property it believes maywhich we believe might be
useful in connection with itsour products. The Company believesWe believe that itsour know-how will also
provide a significant competitive advantage, and intendswe intend to continue to
develop and protect itsour proprietary know-how. The CompanyWe may also from time to time seek
to acquire licenses to important externally developed technologies.
The Company hasWe have exclusive or non-exclusive rights to a portfolio of patents and
patent applications related to various stem and progenitor cells and methods of
deriving and using them. These patents and patent applications relate mainly to
compositions of matter, methods of obtaining such cells, and methods for
preparing, transplanting and utilizing such cells. Currently, the Company'sour U.S. patent
portfolio in the stem cell therapy area includes 16nineteen issued U.S. patents,
with an additional eight patent applications pending, foursix of which have been
allowed and are awaiting issuance.
The patent positions of pharmaceutical and biotechnology companies,
including those ofissued within the Company, are uncertain and involve complex and evolving
legal and factual questions. The coverage sought in a patent application can be
denied or significantly reduced before or after the patent is issued.
Consequently, the Company does not know whether any of its pending applications
will result in the issuance of patents, or if any existing or future patents
will provide significant protection or commercial advantage or will be
circumvented by others. Sincelast year. An additional thirteen patent
applications are secret until patents are
issued in the United Statespending, one of which has been allowed.
We own or until the applications are published in foreign
countries, and since publication of discoveries in the scientific or patent
literature often lags behind actual discoveries, the Company cannot be certain
that it was the first to make the inventions covered by each of its pending
patent applications or that it was the first to file patent applications for
such inventions. There can be no assurance that patents will issue from the
Company's pending or future patent applications or, if issued, that such patents
will be of commercial benefit to the Company, afford the Company adequate
protection from competing products or not be challenged or declared invalid.
In the event that a third party has also filed a patent application relating
to inventions claimed in Company patent applications, the Company may have to
participate in interference proceedings declared by the United States Patent and
Trademark Office to determine priority of invention, which could result in
substantial uncertainties and cost for the Company, even if the eventual outcome
is favorable to the
13
Company. There can be no assurance that the Company's patents, if issued, would
be held valid by a court of competent jurisdiction.
A number of pharmaceutical, biotechnology and other companies, universities
and research institutions have filed patent applications orwhich have been issuedpublished for the
following U.S. patents: Patent Number 5,968,829 (Human CNS neural stem cells);
Patent Number 6,103,530 (Human CNS neural stem cells--culture media);
Application Number WO 99/11758 (Cultures of human CNS neural stem cells); and
Application Number WO 00/36091 (An animal model for identifying a common stem/
progenitor to liver cells and pancreatic cells).
We have licensed the following patents relating toor pending patent applications from
Neurospheres Holdings Ltd.: Patent Number 5,851,832 (In vitro proliferation);
Patent Number 5,750,376 (In vitro genetic modification); Patent
Number 5,981,165 (In vitro production of dopaminergic cells from mammalian
central nervous system multipotent stem cell therapy,compositions); Patent Number
6,093,531 (Generation of hematopoietic cells from multipotent neural stem
cells); Application Number WO 93/01275 (Mammalian central nervous system
multipotent stem cell compositions); Application Number WO 94/09119
(Remyelination using mammalian central nervous system multipotent stem cell
compositions); Application Number WO 94/10292 (Biological factors useful in
differentiating mammalian central nervous system multipotent stem cell
compositions); Application Number WO 94/16718 (Genetically engineered mammalian
central nervous system multipotent stem cell compositions); Application Number
WO 96/15224 (Differentiation of mammalian central nervous system multipotent
stem cell compositions); and Application Number WO 96/15226 (In vitro production
of dopaminergic cells from mammalian central nervous system multipotent stem
cell composition).
We have licensed the following patents or pending patent applications from
the University of California, San Diego: Patent Number 5,776,948 (Method of
production of neuroblasts); Patent Number 6,013,521 (Method of production of
neuroblasts); Patent Number 6,020,197 (Method of production of neuroblasts); and
Application Number WO 94/16059 (Method of production of neuroblasts).
We have licensed the following patents or pending patent applications from
the California Institute of Technology: Patent Number 5,629,159 (Immortalization
and disimmortalization of cells); Application Number WO 96/40877
(Immortalization and disimmortalization of cells); Patent Number 5,935,811
(Neuron restrictive silencer factor proteins); Application Number WO 96/27665
(Neuron restrictive
14
silencer factor proteins); Patent Number 5,589,376 (Mammalian neural crest stem
cells); Patent Number 5,824,489 (Methods for isolating mammalian multipotent
neural crest stem cells); Application Number WO 94/02593 (Mammalian neural crest
stem cells); Patent Number 5,654,183 (Genetically engineered mammalian neural
crest stem cells); Patent Number 5,928,947 (Mammalian multipotent neural crest
stem cells); Patent Number 5,693,482 (In vitro neural crest stem cell assay);
Patent Number 6,001,654 (Methods for differentiating neural stem cells and other technologies potentially
relevant to
neurons or required by the Company's expected products. The Company cannot
predict which, if any, of such applications will issue as patents or the claims
that might be allowed. The Company is aware that a number of companies have
filed applications relating tosmooth muscle cells (TGFb)); Application Number WO 98/48001 (Methods
for differentiating neural stem cells. The Company is also aware of a number
of patent applications and patents claiming use of genetically modified cells to treat disease, disorderneurons or injury. The Company is aware of two patents issuedsmooth muscle cells (TGFb));
Patent Number 5,672,499 (Methods for immortalizing multipotent neural crest stem
cells); Patent Number 5,849,553 (Immortalizing and disimmortalizing multipotent
neural crest stem cells); and Patent Number 6,033,906 (Differentiating mammalian
neural stem cells to a competitor claiming certain methods for treating defective, diseased or
damagedglial cells in the mammalian CNS by grafting genetically modified donor cells
from the same mammalian species.
If third party patents or patent applications contain claims infringed by
the Company's technology and such claims or claims in issued patents are
ultimately determined to be valid, there can be no assurance that the Company
would be able to obtain licenses to these patents at a reasonable cost, if at
all, or be able to develop or obtain alternative technology. If the Company is
unable to obtain such licenses at a reasonable cost, it may be adversely
affected. There can be no assurance that the Company will not be obliged to
defend itself in court against allegations of infringement of third party
patents. Patent litigation is very expensive and could consume substantial
resources and create significant uncertainties. An adverse outcome in such a
suit could subject the Company to significant liabilities to third parties,
require disputed rights to be licensed from third parties, or require the
Company to cease using such technology.
The Companyneuregulins).
We also reliesrely upon trade-secret protection for itsour confidential and
proprietary information. There can be no assurance that others will not
independently develop substantially equivalent proprietary information or
techniques, gainand take active measures to control access to the Company's trade secrets or disclose such
technology, or that
the Company can meaningfully protect its trade secrets.
The Company'sinformation.
Our policy is to require itsour employees, consultants and significant
scientific collaborators and sponsored researchers to execute confidentiality
agreements upon the commencement of an employment or consulting relationship
with the Company.us. These agreements generally provide that all confidential information
developed or made known to the individual by the Companyus during the course of the
individual's relationship with the Companyus is to be kept confidential and not disclosed
to third parties except in specific circumstances. In the case of employees and
consultants, the agreements generally provide that all inventions conceived by
the individual in the course of rendering services to the Companyus shall be theour exclusive
property of the
Company. There can be no assurance, however, that these agreements will provide
meaningful protection or adequate remedies for the Company in the event of
unauthorized use, transfer or disclosure of such information or inventions.
The Company hasproperty.
We have obtained rights from universities and research institutions to
technologies, processes and compounds that it believeswe believe may be important to the
development of itsour products. These agreements typically require the Companyus to pay
license fees, meet certain diligence obligations and, upon commercial
introduction of certain products, pay royalties. These include exclusive license
agreements with NeuroSpheres, The Scripps Institute, the California Institute of
Technology and the Oregon Health Sciences University, to certain patents and
know-how regarding present and certain future developments in neuralCNS and pancreaticpancreas
stem cells.
The Company's licenses may be canceled or converted to
non-exclusive licenses if the Company fails to use the relevant technology or
the Company breaches its agreements. Loss of such licenses could expose the
Company to the risks of third party patents and/or technology. There can be no
assurance that any of these licenses will provide effective protection against
the Company's competitors.
14
COMPETITION
While in some instances theThe targeted disease states for the Company'sour initial products in some instances
currently have no effective long-term therapies, the Company'stherapies. However, we do expect that our
initial products are expectedwill have to compete with a variety of therapeutic products and
procedures. Major pharmaceutical companies currently offer a number of
pharmaceutical products to treat neurodegenerative pancreatic and liver diseases, diabetes
and other diseases for which the Company'sour technologies may be applicable. The Company believesMany
pharmaceutical and biotechnology companies are investigating new drugs and
therapeutic approaches for the same purposes, which may achieve new efficacy
profiles, extend the therapeutic window for such products, alter the prognosis
of these diseases, or prevent their onset. We believe that itsour products, ifwhen
successfully developed, will compete with these products principally on the
basis of improved and extended efficacy and safety and thetheir overall economic
benefit to the health care system offered by such products. However, many other pharmaceutical and
biotechnology companies are investigating new drugs and therapeutic approaches
to treat neurodegenerative, pancreatic and liver diseases, which may achieve new
and better efficacy profiles, extend the therapeutic window for such products,
alter the prognosis of these diseases or prevent their onset.system.
The market for therapeutic products that address degenerative diseases is
large, and competition is intense and is expectedintense. We expect competition to increase. The Company'sWe believe
that our most significant competitors are expected towill be fully integrated pharmaceutical
companies and more established biotechnology companies. Such competitors have
significant resources and expertise in research and development, manufacturing,
testing, obtaining regulatory approvals and marketing and also have
significantly greater capital resources. Smaller companies may
also prove to be significant competitors, particularly through collaborative arrangements
with large pharmaceutical or biotechnology companies. Many of these competitors
have significant products approved or in development that could be competitive
with the Company'sour potential products,products.
Competition for our stem and also operate large, well-funded research
and development programs. In addition, the Company competes with other companies
and institutions for highly qualified scientific and management personnel.
The Company's stem/progenitor cell products if successfully developed,
might face competition from orally administeredmay be in the form of
existing and new drugs, other forms of cell transplantation, ablative and
stimulativesimulative procedures, and gene therapy and new drugs
under development. In addition, the Company believestherapy. We believe that itssome of our competitors
are also trying to develop stem/stem and progenitor cell-based technologies. The Company expectsWe
expect
15
that all of these products if developed, will compete with the Company'sour potential stem/stem and progenitor
cell products based on efficacy, safety, cost and intellectual property
positions.
The Company expects that gene therapy, if successfully
developed, will also be a source of competition for potential stem/progenitor
cell products.
There can be no assurance that the Company's competitors will not succeed in
developing technologies and products that are more effective than those being
developed by the Company or that would render the Company's technology obsolete
or non-competitive. The CompanyWe may also face competition from companies that have filed patent
applications relating to the use of genetically modified cells to treat disease,
disorder or injury. The CompanyWe may be required to seek licenses from these competitors
in order to commercialize certain of itsour proposed products.
Once our products are developed and there can be no assurance that the Company will be able to
obtain such licenses at a reasonable cost, if at all.
Any product that the Company succeeds in developing and for which it gainsreceive regulatory approval, they must
then compete for market acceptance and market share. For certain of the Company'sour
potential products, an important competitivesuccess factor will be the timing of market
introduction of competitive products. Accordingly,
the Company expects that an important competitive factor will beThis is a function of the relative speed
with which the Companywe and itsour competitors can develop products, complete the clinical
testing and approval processes, and supply commercial quantities of a product to
market. With respect toThese competitive products may also impact the timing of clinical
testing competition may delay
progressand approval processes by limiting the number of clinical investigators
and patients available to test the Company'sour potential products.
Competition forWhile we believe that the Company's products is also expected toprimary competitive factors will be based on product
efficacy, safety, and the timing and scope of regulatory approvals, including,other
factors include, in certain instances, obtaining marketing exclusivity under the
Orphan Drug Act, availability of supply, marketing and sales capability,
reimbursement 15
coverage, price, and patent and technology position.
There can be no assurance
that the Company's technology, if fully developed, will become commercially
viable.
GOVERNMENT REGULATION
TheOur research and development activities and the future manufacturing and
marketing of the Company'sour potential products and its
research and development activities are, and will continue to be, subject to
regulation for safety and efficacy by numerous governmental authorities in the
United States and other countries.
In the United States, pharmaceuticals, biologicals and medical devices are
subject to rigorous Food and Drug Administration, or FDA, regulation. The
Federal Food, Drug and Cosmetic Act, as amended, and the Public Health Service
Act, as amended, the regulations promulgated thereunder, and other Federal and
state statutes and regulations govern, among other things, the testing,
manufacture, safety, efficacy, labeling, storage, export, record keeping,
approval, marketing, advertising and promotion of the Company'sour potential products.
Product development and approval within this regulatory framework takes a
number of years and involves substantialsignificant uncertainty combined with the
expenditure of substantial resources. In addition, the United States, statesfederal, state, and other
jurisdictions have restrictions on the use of fetal tissue that could
restrict the Company's use of such materials.tissue.
FDA APPROVAL
The steps required before the Company'sour potential products may be marketed in the
United States include (i) preclinical laboratory and animal tests,
(ii) the submission to the FDA of an application for an Investigational New Drug
Exemption ("IND"), which must become effective before U.S. human clinical trials
may commence, (iii) adequate and well-controlled human clinical trials to
establish the safety and efficacy of the product, (iv) the submission to the FDA
of a marketing authorization application(s) and (v) FDA approval of the
application(s) prior to any commercial sale or shipment of the drug. Biologic
product manufacturing establishments located in certain states also may be
subject to separate regulatory and licensing requirements.
Preclinical tests include laboratory evaluation of the product and animal
studies to assess the potential safety and efficacy of the product and its
formulation as well as the quality and consistency of the manufacturing process.
The results of the preclinical tests are submitted to the FDA as part of an IND,
and the IND becomes effective 30 days following its receipt by the FDA, absentinclude:
STEPS CONSIDERATIONS
1. Preclinical laboratory and animal tests Preclinical tests include laboratory
evaluation of the product and animal studies
in specific disease models to assess the
potential safety and efficacy of the product
and our formulation as well as the quality
and consistency of the manufacturing process.
2. Submission to the FDA of an application The results of the preclinical tests are
for an Investigational New Drug Exemption, or submitted to the FDA as part of an IND, and
IND, which must become effective before U.S. the IND becomes effective 30 days following
human clinical trials may commence its receipt by the FDA, as long as there are
no questions, requests for delay or
objections from the FDA.
Clinical trials involve the evaluation of the product in healthy volunteers
or, as may be the case with the Company's potential products, in a small number
of patients under the supervision of a qualified physician. Clinical trials are
conducted in accordance with protocols that detail the objectives of the study,
the parameters to be used to monitor safety and the efficacy criteria to be
evaluated. Any product administered in a U.S. clinical trial must be
manufactured in accordance with clinical Good Manufacturing Practices ("cGMP")
determined by the FDA. Each protocol is submitted to the FDA as part of the IND.
The protocol for each clinical study must be approved by an independent
Institutional Review Board ("IRB") at the institution at which the study is
conducted and the informed consent of all participants must be obtained. The IRB
will consider, among other things, the existing information on the product,
ethical factors, the safety of human subjects, the potential benefits of therapy
and the possible liability of the institution.
Clinical development is traditionally conducted in three sequential phases.
The phases may overlap, however. In Phase I, products are typically introduced
into healthy human subjects or into selected patient populations to test for
safety (adverse reactions), dosage tolerance, absorption and distribution,
metabolism, excretion and clinical pharmacology. Phase II involves studies in a
limited patient population to (i) determine the efficacy of the product for
specific targeted indications and populations, (ii) determine optimal dosage and
dosage tolerance and (iii) identify possible adverse effects and safety risks.
When a dose is chosen and a candidate product is found to be effective and to
have an acceptable safety profile in Phase II evaluations, Phase III trials are
undertaken to conclusively demonstrate clinical efficacy and to test further for
safety within an expanded patient population, generally at multiple study sites.
The FDA
16
continually reviews the clinical trial plans and results and may suggest changes
or may require discontinuance of the trials at any time if significant safety
issues arise. The results of the preclinical studies and clinical studies are
submitted to the FDA in the form of a marketing approval authorization
application.
The testing and approval process is likely to require substantial time,
effort and expense and there can be no assurance that any FDA approval will be
granted on a timely basis, if at all. The time to approval is affected by a
number of factors, including relative risks and benefits demonstrated in
clinical trials, the availability of alternative treatments and the severity of
the disease. Additional animal studies or clinical trials may be requested
during the FDA review period and may delay marketing approval.
3. Adequate and well-controlled human Clinical trials involve the evaluation of the
clinical trials to establish the safety and product in healthy volunteers or, as may be
efficacy of the product the case with our potential products, in a
small number of patients under the
supervision of a qualified physician.
Clinical trials are conducted in accordance
with protocols that detail the objectives of
the study, the parameters to be used to
monitor safety and the efficacy criteria to
be evaluated. Any product administered in a
U.S. clinical trial must be manufactured in
accordance with clinical Good Manufacturing
Practices, or cGMP, determined by the FDA.
Each protocol is submitted to the FDA as part
of the IND. The protocol for each clinical
study must be approved by an independent
Institutional Review Board, or IRB, at the
institution at which the study is conducted
and the informed consent of all participants
must be obtained. The IRB will consider,
among other things, the existing information
on the product, ethical factors, the safety
of human subjects, the potential benefits of
the therapy and the possible liability of the
institution.
Clinical development is traditionally
conducted in three sequential phases, which
may overlap:
- In Phase I, products are typically
introduced into healthy human subjects or
into selected patient populations to test for
adverse reactions, dosage tolerance,
absorption and distribution, metabolism,
excretion and clinical pharmacology.
- Phase II involves studies in a limited
patient population to (i) determine the
efficacy of the product for specific targeted
indications and populations, (ii) determine
optimal dosage and dosage tolerance and (iii)
identify possible adverse effects and safety
risks. When a dose is chosen and a candidate
product is found to be effective and to have
an acceptable safety profile in Phase II
evaluations, Phase III trials begin.
- Phase III trials are undertaken to
conclusively demonstrate clinical efficacy
and to test further for safety within an
expanded patient population, generally at
multiple study sites.
The FDA continually reviews the clinical
trial plans and results and may suggest
changes or may require discontinuance of the
trials at any time if significant safety
issues arise.
4. Submission to the FDA of marketing The results of the preclinical studies and
authorization applications clinical studies are submitted to the FDA in
the form of marketing approval authorization
applications.
17
5. FDA approval of the application(s) prior The testing and approval process will require
to any commercial sale or shipment of the substantial time, effort and expense. The
drug. Biologic product manufacturing time for approval is affected by a number of
establishments located in certain states also factors, including relative risks and
may be subject to separate regulatory and benefits demonstrated in clinical trials, the
licensing requirement availability of alternative treatments and
the severity of the disease. Additional
animal studies or clinical trials may be
requested during the FDA review period which
might add to that time.
After FDA approval for the initial indications and requisite approval of the
manufacturing facility, further clinical trials may be necessaryrequired to gain approval
for the use of the product for additional indications. The FDA may also require
unusual or restrictive post-marketing testing and surveillance to monitor for
adverse effects, which cancould involve significant expense, or may elect to grant
only conditional approvals.
FDA MANUFACTURING REQUIREMENTS
Among the conditions for product licensure is the requirement that the
prospective manufacturer's quality control and manufacturing procedures conform
to cGMP.the FDA's cGMP requirement. Even after product licensure approval, the
manufacturer must comply with cGMP on a continuing basis, and what constitutes
cGMP may change as the state of the art of manufacturing changes. Domestic
manufacturing facilities are subject to regular FDA inspections for cGMP
compliance (normallywhich are normally held at least every two years), and foreignyears. Foreign
manufacturing facilities are subject to periodic FDA inspections or inspections
by the foreign regulatory authorities with reciprocal inspection agreements with
the FDA. Domestic manufacturing facilities may also be subject to inspection by
foreign authorities.
ORPHAN DRUG ACT
The Orphan Drug Act provides incentives to drug manufacturers to develop and
manufacture drugs for the treatment of diseases or conditions that affect fewer
than 200,000 individuals in the United States. Orphan drug status can also be
sought for treatments for diseases or conditions that affect more than 200,000
individuals in the United States if the sponsor does not realistically
anticipate its product becoming profitable from sales in the United States. The
CompanyWe
may apply for orphan drug status for certain of itsour therapies.
Under the Orphan Drug Act, a manufacturer of a designated orphan product can
seek tax benefits, and the holder of the first FDA approval of a designated
orphan product will be granted a seven-year period of marketing exclusivity in
the United States for that product for the orphan indication. While the
marketing exclusivity of an orphan drug would prevent other sponsors from
obtaining approval of the same compound for the same indication, it would not
prevent other types of products from being approved for the same use including,
in some cases, slight variations on the originally designated orphan product.
Legislation has been introduced in the U.S. Congress in the past, and is likely
to be introduced again in the future, that would restrict the extent and
duration of the market exclusivity of an orphan drug, and there can be no
assurance that the benefits of the existing statute will remain in effect.PROPOSED FDA REGULATIONS
Proposed regulations of the FDA and other governmental agencies would place
restrictions, including disclosure requirements, on researchers who have a
financial interest in the outcome of their research. Under the proposed
regulations, the FDA could also apply heightened scrutiny to, or exclude the
results of, studies conducted by such researchers when reviewing applications to
the FDA, which contain such research. Certain of the Company'sour collaborators have stock
options or other equity interests in the Companyus that could subject such collaborators
and the Companyus to the proposed regulations.
Our research and development is based on the use of human stem and
progenitor cells. The FDA has published a "Proposed Approach to Regulation of
Cellular and Tissue-Based Products" which relates to the use of human cells. The CompanyWe
cannot presentlynow determine the effects of that approach or what other regulatory actions
might be taken.taken from it. Restrictions exist on the testing or use of cells,
whether human or non-human, as human therapeutics could adversely affect the Company's
product development programs and the Company itself and could prevent the
Company from producing and/or selling certain products or make the cost of
production by the Company prohibitively high.non-human.
18
OTHER REGULATIONS
In addition to safety regulations enforced by the FDA, the Company iswe are also subject
to regulations under the Occupational Safety and Health Act, the Environmental
Protection Act, the Toxic Substances 17
Control Act and other present and potential
future supranational, foreign, Federal, state and local regulations.
Outside the United States, the Companywe will be subject to regulations which govern
the import of drug products from the United States or other manufacturing sites
and foreign regulatory requirements governing human clinical trials and
marketing approval for itsour products. The requirements governing the conduct of
clinical trials, product licensing, pricing and reimbursements vary widely from
country to country. In particular, the European Union, ("EU")or EU, is revising its
regulatory approach to high tech products, and representatives from the United
States, Japan and the EU are in the process orof harmonizing and making more
uniform the regulations for the registration of pharmaceutical products in these
three markets.
Although certain of such proposals have been adopted, the Company
cannot anticipate what effect the adoption of the final forms of this
harmonization or the changes to the EU high tech procedure may have.
REIMBURSEMENT AND HEALTH CARE COST CONTROL
The Company's ability to commercialize products successfully may depend in
part on the extent to which reimbursementReimbursement for the costs of treatments and products such products and
related treatments will be availableas ours from
government health administration authorities, private health insurers and others
both in the United States and abroad.abroad is a key element in the success of new
health care products. Significant uncertainty often exists as to the
reimbursement status of newly approved health care products.
Reimbursement limitations or prohibitions with
respect to any product developedThe revenues and profitability of some health care-related companies have
been affected by the Company could adversely affect the
Company's ability to establishcontinuing efforts of governmental and maintain price levels sufficient for
realization of an appropriate return on its investment in developing new
therapies. Government and other third party payorspayers
to contain or reduce the cost of health care through various means. Payers are
increasingly attempting to contain health care costs by limitinglimit both coverage and the level of reimbursement
for new therapeutic products approved for marketing by the FDA, and byare
refusing, in some cases, to provide any coverage for uses of approved products
for disease indications for which the FDA has not granted marketing approval.
If adequate coverage and reimbursement levels are not provided by
third party payors for uses of the Company's therapeutic products, the market
acceptance of these products would be adversely affected.
The revenues and profitability of health care-related companies may be
affected by the continuing efforts of governmental and third party payers to
contain or reduce the cost of health care through various means. For example, in certain foreign markets, pricing or profitability of
prescription pharmaceuticals is subject to government control. In the United
States, there have been and the
Company expects that there will continue to be, a number of Federal and state proposals to implement
government control over health care costs.
Efforts at
healthcare reform are likely to continue in future legislative sessions. It is
uncertain what legislative proposals will be adopted or what actions Federal,
state or private payers for healthcare goods and services may take in response
to healthcare reform proposalsEMPLOYEES
As of legislation. The Company cannot predict the
effect healthcare reforms mayAugust 15, 2000, we had twenty full-time employees, of whom five have
on its business. Any such reformsPh.D. degrees, as well as the uncertainty their proposal engenders could have a material adverse effect on
the Company.
EMPLOYEES
Astwo half-time employees. The equivalent of March 20, 1999, the Company had 21 full- and part-time employees
(including two contract employees), of whom four have Ph.D. degrees.
Approximately 14fifteen
full-time employees work in research and development and laboratory support
services. A number of the Company'sour employees have held positions with other biotechnology
or pharmaceutical companies or have worked in university research programs. No
employees are covered by collective bargaining agreements.
SCIENTIFIC ADVISORY BOARD
Members of the Company'sour Scientific Advisory Board provide the Companyus with strategic guidance
in regard to itsour research and product development programs, as well as
assistance in recruiting employees and 18
collaborators. Each Scientific Advisory
Board member has entered into a consulting agreement with the Company.us. These consulting
agreements typically specify the compensation to be paid to the consultant and require
that all information about the Company'sour products and technology be kept confidential. All
of the Scientific Advisory Board members are employed by employers other than the Companyus
and may have commitments to or consulting or advising agreements with other
entities which maythat limit their availability to the
Company.us. The Scientific Advisory Board
members have generally agreed, however, for so long as they serve as consultants
to the Company,us, not to provide any services to any other entities which would conflict
with the services the member provides to the Company.us. Members of the Scientific Advisory
Board offer consultation on specific issues encountered by the Companyus as well as general
advice on the directions of appropriate scientific inquiry for the Company.us. In addition, certain
Scientific Advisory Board members assist the Companyus
19
in assessing the appropriateness of moving the Company'sour projects to more advanced stages.
The following persons are members of the Company'sour Scientific Advisory Board:
- Irving L. Weissman, M.D., is the Karel and Avice Beekhuis Professor of
Cancer Biology, Professor of Pathology and Professor of Developmental
Biology at Stanford University. Dr. Weissman iswas a cofounder of
SyStemix, Inc., and Chairman of theits Scientific Advisory Board of
SyStemix, Inc.Board. He has
served on the Scientific Advisory Boards of Amgen Inc., DNAX and T-Cell
Sciences, Inc. Dr. Weissman is Chairman of the Scientific Advisory Board
of CytoTherapeutics.StemCells, Inc.
- David J. Anderson, Ph.D., is Professor of Biology, California Institute of
Technology, Pasadena, California and Investigator, Howard Hughes Medical
Institute.
- Fred H. Gage, Ph.D., is Professor, Laboratory of Genetics, The Salk
Institute for Biological Studies, La Jolla, California and Adjunct
Professor, Department of Neurosciences, University of California, San
Diego, California.
ITEM 2. PROPERTIES
The Company's current research laboratories and administrative offices are
located in a leased 7,950 square-foot multipurpose building housing wet labs,
specialty research areas and administrative offices located in Sunnyvale,
California. The facilities are leased pursuant to lease agreements expiring
August 31, 2001, with the Company havingand we have certain renewal options. The Company'sOur current facilities are
expected to be sufficient to accommodate itsour needs at least through the end of
2000.
The Company continuesWe continue to be the lessee of the followingcontractually obligated with respect to two facilities in
Lincoln, Rhode Island obtained in connection with itsour former encapsulated cell
technology: itstechnology. We leased our former research laboratory and corporate headquarters
building which contains 65,000 square feet of wet labs, specialty research areas
and administrative offices held on a fifteen-year lease agreement as well aswhich expires
October 2012. We also own a 21,000 square-foot pilot manufacturing facility and
a 3,000 square-foot cell processing facility financed by bonds issued by the
Rhode Island Industrial Facilities Corporation. The Company isWe are actively seeking to
sublease, assign or sell itsour interests in these properties.
ITEM 3. LEGAL PROCEEDINGS
None.
ITEM 4. SUBMISSION OF MATTERS TO A VOTE OF SECURITY HOLDERS
None.
1920
PART II
ITEM 5. MARKET FOR REGISTRANT'S COMMON EQUITY AND RELATED STOCKHOLDERS MATTERS
The Common Stock of CytoTherapeutics is traded on the National Market System
of NASDAQ under the Symbol CTII.STEM (formerly CTII). The quarterly ranges of high
and low sales prices since January 1, 1997 are shown below:
2000 HIGH LOW
- ---- ------------- ------------
First Quarter (through March 20, 2000)...................... $20 $1 3/8
1999 HIGH LOW
- ------------------------------------------------------------ ------------- ------------
Fourth Quarter.............................................. $ 1 5/8 $1
Third Quarter............................................... $ 2 3/8 $ 11/16
Second Quarter.............................................. $ 1 3/8 $ 17/32
First Quarter............................................... $ 1 25/32 $1 5/32
1998 HIGH LOW
- ------------------------------------------------------------ ------------- ------------
Fourth Quarter.............................................. $ 2 17/32 $ 26/32
Third Quarter............................................... $ 1 19/32 $ 29/32
Second Quarter.............................................. $ 3 7/16 $1 1/16
First Quarter............................................... $ 4 3/8 $2 1/2
1997 HIGH LOW
- ------------------------------------------------------------ ------------- ------------
Fourth Quarter.............................................. $ 7 5/8 $3 7/16
Third Quarter............................................... $ 6 1/4 $4 5/8
Second Quarter.............................................. $ 8 3/4 $4 3/4
First Quarter............................................... $11 3/8 $7 1/2
No cash dividends have been declared on the Common Stock since the Company's
inception.
As of March 20,August 15, 2000, there were approximately 279249 holders of record of the
Common Stock.
2021
ITEM 6. SELECTED FINANCIAL DATA
YEAR ENDED DECEMBER 31,
----------------------------------------------------
1999 1998 1997 1996 1995
-------- -------- -------- -------- --------
(IN THOUSANDS, EXCEPT PER SHARE AMOUNTS)
STATEMENT OF OPERATIONS DATA
Revenue from collaborative agreements....... $ 5,022 $ 8,803 $ 10,617 $ 7,104 $11,761
Research and development expenses........... 9,991 17,659 18,604 17,130 14,730
Acquired research and development........... 8,344
Wind-down expenses..........................ECT wind-down expenses...................... 6,048
Net loss.................................... (15,709) (12,628) (18,114) (13,759) (8,891)
Basic and diluted net loss per share........ (0.84) (0.69) (1.08) (0.89) (0.69)
Shares used in computing basic and diluted
net loss per share........................ 18,706 18,291 16,704 15,430 12,799
DECEMBER 31,
----------------------------------------------------
1999 1998 1997 1996 1995
-------- -------- -------- -------- --------
(IN THOUSANDS)
BALANCE SHEET DATA
Cash, cash equivalents and marketable
securities................................ $ 4,760 $ 17,386 $ 29,050 $ 42,607 $44,192
Total assets................................ 16,08115,781 32,866 44,301 58,397 56,808
Long-term debt, including capitalized
leases.................................... 2,937 3,762 4,108 8,223 5,441
Redeemable common stock..................... 5,249 5,249 5,583 8,159
Stockholders' equity........................ 3,506 17,897 28,900 34,747 45,391
2122
\
ITEM 7. MANAGEMENT'S7.MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS
OF OPERATIONS
The following discussion of theour financial condition and results of
operations of CytoTherapeutics, Inc.for the years ended December 31, 1999, 1998, and 1997 should be read
in conjunction with the
accompanyingour consolidated financial statements and thetheir related
footnotes thereto.footnotes.
The statements contained in this report, other than statements of historical
fact, constitute forward-looking statements. Such statements include, without
limitation, all statements as to expectation or belief and statements as to the
Company's future results of operations, the progress of the Company's research
and product development programs, the need for, and timing of, additional
capital and capital expenditures, partnering prospects, the need for additional
intellectual property rights, effects of regulations, the need for additional
facilities and potential market opportunities. The Company's actual results may
vary materially from those contained in such forward-looking statements because
of risks to which the Company is subject, such as failure to obtain a corporate
partner or partners to support the development of the Company's stem cell
programs, the Company's ability to sell, assign or sublease its interest in its
facilities related to its encapsulated cell technology program, risks of delays
in research, development and clinical testing programs, obsolescence of the
Company's technology, lack of available funding, competition from third parties,
intellectual property rights of third parties, failure of the Company's
collaborators to perform, regulatory constraints, litigation and other risks to
which the Company is subject. See "Cautionary Factors Relevant to
Forward-Looking-Information" filed herewith as Exhibit 99 and incorporated
herein by reference.
RESULTS OF OPERATIONS
OVERVIEW
Since itsour inception in August 1988 the Company haswe have been primarily engaged in research and
development of human therapeutic products. NoAs a result of a restructuring in the
second half of 1999, our sole focus is now on our stem cell technology. At the
beginning of last year, by contrast, our corporate headquarters, most of our
employees, and the main focus of our operations were primarily devoted to a
different technology--encapsulated cell technology, or ECT. Since that time, we
terminated a clinical trial of the ECT then in progress, we wound down our other
operations relating to the ECT, we terminated the employment of those who worked
on the ECT, sold the ECT and we relocated from Rhode Island to Sunnyvale,
California. Comparisons with last year's results are correspondingly less
meaningful than they may be under other circumstances.
We were known as CytoTherapeutics, Inc., until May 23, 2000, when we changed
our name to StemCells, Inc.
We have not derived any revenues have been
derived from the sale of any products, and the Company doeswe do
not expect to receive revenues from product sales for at least several years. The Company hasWe
have not commercialized any product and in order to do so itwe must, among other
things, substantially increase itsour research and development expenditures as
research and product development efforts accelerate and clinical trials are
initiated. The Company hasWe have incurred annual operating losses since inception and expectsexpect
to incur substantial operating losses in the future. As a result, the
Company iswe are
dependent upon external financing from equity and debt offerings and revenues
from collaborative research arrangements with corporate sponsors to finance itsour
operations. There are no such collaborative research arrangements at this time
and there can be no assurance that such financing or partnering revenues will be
available when needed or on terms acceptable to the
Company. The Company'sus.
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, nonrecurring events, including without limitation the
receipt of one-time, nonrecurring licensing payments, and the initiation or
termination of
23
research collaborations, andin addition to the winding downwinding-down of terminated research
and development programs.
RESULTS OF OPERATIONSprograms referred to above.
YEARS ENDED DECEMBER 31, 1999, 1998 AND 1997
Revenues from collaborative agreements totaled $5,022,000, $8,803,000 and
$10,617,000 for the years ending December 31, 1999, 1998 and 1997, respectively.
Revenues were earned primarily from a Development, Marketing and License
Agreement with AstraZeneca Group plc, which waswe signed in March 1995 (the "Astra
Agreement").1995. The
decrease in revenues from 1998 to 1999 resulted primarily from the June 1999
termination of the AstraAstraZeneca Agreement. 1997 revenues included a $3,000,000
milestone payment from AstraAstraZeneca related to the Phase II clinical programtrials for
the Company's pain control implant.
22
an ECT product.
Research and development expenses totaled $9,984,000 in 1999, as compared to
$17,659,000 in 1998 and $18,604,000 in 1997. The decrease of $7,668,000, or 43%,
from 1998 to 1999 was primarily attributable to the wind-down of research
activities relating to the Company's encapsulated cell technology,ECT, precipitated by termination of the Astra Agreement.AstraZeneca
agreement. The decrease of $945,000, or 5%, from 1997 to 1998 was primarily
attributable to a reduction in spending on research agreements and a reduction
in research and development personnel.
Acquired research and development consists of a one-time charge of
$8,344,000 related to the acquisition of StemCells California, Inc., in 1997.
Commercialization of this technology will require significant incremental
research and development expenses over a number of years. With the recent
completion of the restructuring of the Company'sour research operations, the
Company iswe are now focused
solely on the research and development of itsour platform of stem cell
technology,technologies, which encompasses the technology acquired upon the acquisition of
StemCells California, Inc. and related technology we have developed or licensed in by the Company.licensed.
General and administrative expenses were $4,927,303 for the year ended
December 31, 1999, compared with $4,603,000 in 1998 and $6,158,000 in 1997. DueThe
1999 General and Administrative expenses were positively impacted by the
reduction in facility costs that were included in the wind-down ($239,000),
reduction in amortization of patents and intangible assets of approximately
$338,000, as well as reduced activities and related personnel costs estimated at
approximately $500,000 that were not incurred. This was due to the wind-down of
the Company's encapsulated cell technologyour ECT programs and relocation of the Company'sour headquarters in October the 1999 expenses are less than they
would have been had these events not occurred.1999. The
reduction of $1,555,000, or 25%, from 1997 to 1998 was primarily attributable to
a reduction in legal fees, recruiting and relocation expenses, as well as a
reduction in employees.
Wind-down and relocation expenses totaled $6,047,806 for the year ended
December 31, 1999; no such expenses were incurred in 1998 and 1997. These
expenses relate to the wind-down of the Company'sour encapsulated cell technology research
and the
Company's other Rhode Island operations and the transfer of the Company'sour corporate headquarters
to Sunnyvale, CaliforniaCalifornia.
They include accruals of approximately $1,554,000 for employee severance
costs, $1,858,000 in losses and an accrualreserves for the Company'swrite-down of related patents
and fixed assets, $1,172,000 for our estimate of the costs of settlement of a
1989 funding agreement with the Rhode Island Partnership for Science and
Technology, ("RIPSAT").$702,000 of estimated additional carrying costs through an expected
June 30, 2000 disposition of the Rhode Island facilities, and other related
expenses totaling $762,000.
Interest income for the years ended December 31, 1999, 1998 and 1997 totaled
$564,000, $1,254,000 and $1,931,000, respectively. The average cash and
investment balances were $10,663,000, $21,795,000 and $33,343,000 in 1999, 1998
and 1997, respectively. The decrease in interest income from 1997 to 1998 to
1999 was attributable to lower average balances.
In 1999, interest expense was $335,000, compared with $472,000 in 1998 and
$438,000 in 1997. The decrease from 1998 to 1999 was attributable to lower
outstanding debt and capital lease balances. The increase from 1997 to 1998 was
primarily attributable to capitalization of $210,000 of interest on the new
facility in 1997.
24
In October 1997, the Companywe recognized a gain in the amount of $3,387,000 related to
the sale of 50 percent of the Company's interest in Modex Therapeutiques.Therapeutics, Ltd.
The net loss in 1999, 1998 and 1997 was $15,709,000, $12,628,000, and
$18,114,000, respectively. The loss per share was $0.84, $.69$0.69 and $1.08 in
1999, 1998 and 1997, respectively. The increase from 1998 to 1999 is primarily
attributable to the elimination of revenue from the Astra Agreement,AstraZeneca agreement, which
was terminated in June 1999, as well as expenses related to the wind-down of the
Company's encapsulated cell technology researchand itsour
ECT research and our other Rhode Island operations, the transfer of the Company'sour
corporate headquarters to Sunnyvale, California and an accrual of approximately
$1,172,000 for the Company'sour estimate of the costs of settlement of athe funding agreement
with RIPSAT. The decrease from 1997 to 1998 was attributable to a one-time
charge of $8,344,000 for acquired research and development related to the
purchase of StemCells California, Inc. offset by athe $3,387,000 gain on a
partial sale of the Company's interest in Modex in 1997.
23
The 1999 decrease in patents of $3,229,932 from 1998 was primarily due to
management's decision to wind down the ECT program and dispose of the related
intellectual property. During the fourth quarter of 1999 we sold the patents
related to our encapsulated cell technology to Neurotech for $3,000,000.
Accrued expenses increased by $1,584,949, primarily due to the accrual of
approximately $1,172,000 for our estimate of the costs of settlement of a 1989
funding agreement with the Rhode Island Partnership for Science and Technology
and $463,000 for the estimated lease payments and operating costs of the Rhode
Island facilities through an expected disposal date of June 30, 2000.
LIQUIDITY AND CAPITAL RESOURCES
Since itsour inception, the Company haswe have financed itsour operations through the sale of
common and preferred stock, the issuance of long-term debt and capitalized lease
obligations, revenues from collaborative agreements, research grants and
interest income.
The CompanyWe had unrestricted cash and cash equivalents totaling $4,760,000 at
December 31, 1999. Cash and cash equivalents are invested in money market accountsfunds.
We also hold 126,193 shares of Modex stock, which is publicly traded on the
Swiss Neue Market exchange. While our Modex stock had an estimated fair market
value of $27,204,333 on September 30, 2000 (and $23,128,598 on October 31,
2000), the fair market value of our Modex stock has varied significantly since
the Modex public offering and may continue to vary significantly based on
increases and decreases in institutions insured by the FDIC.
The Company'sreported per share price, in Swiss francs, of the
Modex stock and on foreign currency exchange rates. We are prohibited under a
lock-up agreement entered into at the time of Modex's public offering from
selling any of our Modex shares until December 23, 2000. In addition, there is a
limited trading market for Modex stock, and if we were to attempt to sell any
significant portion of our Modex holdings, we would likely be able to do so only
at a significant discount to the then market price, if at all.
Our liquidity and capital resources have been and will continue to
bewere in the past significantly affected
by the Company's relationshipour relationships with corporate partners.partners, which were related to our former
ECT. These relationships are now terminated, and we have not yet established
corporate partnerships with respect to our stem cell technologies.
In March 1995, the Companywe signed a collaborative research and development agreement
with AstraZeneca plc for the development and marketing of certain
encapsulated-cell products to treat pain. AstraZeneca made an initial,
nonrefundable payment of $5,000,000, included in revenue from collaborative
agreements in 1995, a milestone payment of $3,000,000 in 1997 and was to remit
up to an additional $13,000,000 subject to achievement of certain development
milestones. Under the agreement, the Company waswe were obligated to conduct certain research
and development pursuant to a four-year research plan agreed upon by the
parties. Over the term of the research plan, the Companywe originally expected to receive
annual payments of $5 million to $7 million from AstraZeneca, which was to
approximate the research and development costs we incurred by the Company under the plan.
Subject to the successful development of such products and obtaining necessary
regulatory approvals, AstraZeneca was obligated to conduct all clinical trials
of products arising from the collaboration and to
25
seek approval for their sale and use. AstraZeneca had the exclusive worldwide
right to market products covered by the agreement. Until the later of either the
expiration of all patents included in the licensed technology or a specified
fixed term, the Company waswe were entitled to a royalty on the worldwide net sales of such
products in return for the marketing license granted to AstraZeneca and the
Company'sour
obligation to manufacture and supply products. AstraZeneca had the right to
terminate the original agreement beginning April 1, 1998. On June 24, 1999,
AstraZeneca informed the Companyus of the results of AstraZeneca'stheir analysis of the double-blind,
placebo-controlled trial of the Company'sa potential ECT product, an encapsulated bovine cell
implant for the treatment of severe, chronic pain in cancer patients.
AstraZeneca determined that, based on criteria it established, the results from
the 85-patient trial did not meet the minimum statistical significance for
efficacy established as a basis for continuing worldwide trials for the therapy.
AstraZeneca therefore indicated that it did not intend to further develop the
bovine cell-containing implant therapy and exercised its right to terminate the
agreement. (SEE ALSO NOTE 15--(See also Note 16 --"RESEARCH AGREEMENTS" TO
THE ACCOMPANYING FINANCIAL STATEMENTS)Research Agreements" to the Accompanying
Consolidated Financial Statements.)
In the third quarter of 1999, the Companywe announced restructuring plans for the
wind-down of operations relating to its encapsulated cell technologyour ECT and to focus itsour resources on the
research and development of itsour platform of proprietary stem cell technology platform. The Companytechnologies.
We terminated approximately 68 full time employees and, in October 1999,
relocated itsour corporate headquarters to Sunnyvale, California. The CompanyWe recorded
approximately $5.7 million of$6,047,806 in wind-down expenses including employee separation and relocation
costs during 1999.
On December 30, 1999 the Companywe sold its encapsulated cell technology
("ECT")our ECT and assigned our intellectual property
assets in it to Neurotech S.A. for a payment of $3,000,000, royalties on future
product sales, and a portion of certain Neurotech revenues from third parties in
return for the assignment to Neurotech of intellectual property assets relating
to ECT.parties.
In addition, the Companywe retained certain non-exclusive rights to use ECT in combination
with itsour proprietary stem cell technologytechnologies and in the field of vaccines for
prevention and treatment of infectious diseases. The CompanyWe received $2,800,000 of the
initial payment on January 3, 2000 with a remaining balance of $200,000 placed
in escrow, to be received by the Companyreleased to us upon demonstration satisfactory to Neurotech
that certain intellectual property is not subject to other claims.
As part of the Company'sour restructuring of its operations and relocation of
its corporate
headquarters to Sunnyvale, California, the Companywe identified a significant amount of
excess fixed assets. In December 24
of 1999, the Companywe completed the disposition of those
excess fixed assets, from which itwe received more than $746,000. TheseThe proceeds are
expected to bebeing used to fund the Company'sour continuing operations.
In July 1999, as a result of our decision to close our Rhode Island
Facilities, the Rhode Island Partnership for Science and Technology, ("RIPSAT")or RIPSAT,
alleged that the Company waswe were in default under a June, 1989 Funding Agreement (the "Funding Agreement"), and
demanded payment of approximately $2.6 million. While the Company believes it waswe believed we were not in
default under the Funding Agreement, the Companywe deemed it best to resolve the dispute
without litigation, and on March 3, 2000 entered into a settlement agreement
with RIPSAT, the Rhode Island Industrial Recreational Building Authority, ("IRBA")or
IRBA, and the Rhode Island Industrial Facilities Corporation, ("RIIFC"). The Companyor RIIFC. We
agreed to pay RIPSAT $1,172,000 in full satisfaction of all of our obligations
of the Company to RIPSATthem under the Funding Agreement. At the same time, IRBA agreed to return to
the Companyus the full amount of the Company'sour debt service reserve, ("Reserve
Funds"), comprising approximately
$610,000 of principal and interest relating to the bonds the Company haswe had with IRBA and
RIIFC. The Reserve Funds were$610,000 debt service reserve was transferred directly to RIPSAT,
soleaving the net cashremainder of approximately $562,000 to be paid by the Company was
approximately $562,000. The Companyus. We made this
payment in March of 2000.
The Company'sOur liquidity and capital resources could have also been affected by a claim
by Genentech, Inc., arising out of the their collaborative development and licensing
agreement with us relating to the development of products for the treatment of
Parkinson's disease; in the event, however, the claim was resolved with no effect on the Company'sour
resources. On May 21, 1998 Genentech exercised its right to terminate the
Parkinson's collaboration and demanded that the Companywe redeem for approximately $3,100,000, certain shares of the
Company'sour
redeemable Common Stockcommon stock held by Genentech.Genentech for approximately $3,100,000.
Genentech's claim was based on provisions in the agreement requiring the Companyus to
redeem, at the price of $10.01 per share, the shares representing the difference
between the funds invested by Genentech to acquire such stock, and the amount
expended by the
Companyus on the terminated program less an
26
additional $1,000,000. In March 2000, the Company and Genentechwe entered into a Settlement Agreement
with Genentech under which Genentech released the Companyus from any obligation to redeem
any shares of the
Company's Common Stockour common stock held by Genentech, without cost to the Company.us.
Accordingly, the $5.2 million of redeemable common stock shown as a liability in
the Company's December 31, 1999 balance sheet will bewas transferred to equity in
March, 2000, and use of the Company'swithout any impact on our liquidity and capital resources will not be
necessary. The Companyresources. We and
Genentech also agreed that all collaborations between themus were terminated, and
that neither of us had any rights to the intellectual property of the other.
The Company continuesIn May 1996, we secured an equipment loan facility with a bank in the amount
of $2,000,000. On August 5, 1999 we made a payment of approximately $752,000 of
principal and interest to the bank to retire this loan facility rather than seek
a waiver by the bank of our violation of a loan covenant requiring us to
maintain unrestricted liquidity in an amount equal to or in excess of $10
million.
We continue to have substantial outstanding obligations in regard to itsour former
facilities in Lincoln, Rhode Island, including lease payments and operating
costs of approximately $950,000 per year associated with itsour former research
laboratory and corporate headquarters building, and debt service payments and
operating costs of approximately $1,000,000 per year with respect to itsour pilot
manufacturing and cell processing facility. The Company isWe are actively seeking to sublease,
assign or sell itsour interests in these facilities, but there
can be no assurance that the Company will succeed in these effortsfacilities. Failure to do so within a
reasonable period of time period; if it does not, this will have a material adverse effect on the Company'sour liquidity
and capital resources.
In May 1996, the Company secured an equipment loan facility with a bank (the
"Lender") in the amount of $2,000,000 (the "Credit Facility"). On August 5, 1999
the Company made a payment of approximately $752,000 of principal and interest
to the Lender to retire the Credit Facility rather than seek a waiver by the
Lender of the Company's violation of a loan covenant requiring the Company to
maintain unrestricted liquidity in an amount equal to or in excess of
$10 million.
On April 13, 2000 the Company completed arrangements to sellwe sold 1,500 shares of our 6% cumulative convertible
preferred stock plus a warrant for 75,000 shares of the Company'sour common stock to a membertwo
members of itsour Board of Directors for $1,500,000, on terms more favorable to us
than it waswe were able to obtain from outside investors. The face value of the shares
areof preferred stock is convertible at the option of the holderholders into common stock
at a price
to be determined by reference to the price of the Company's common stock for a
period approximately from
25
April 12, 2000 through the twentieth trading day following the filing of this
Form 10-K.$3.77 per share. The conversion price may be below the trading market price of the
stock at the time of conversion. The holderholders of the preferred stock hashave liquidation rights
equal to histheir original investment plus accrued but unpaid dividends. The
investorinvestors would be entitled to make additional investments in the
Companyour securities on
the same terms as those on which the Company completeswe complete offerings of itsour securities with
third parties within 6 months, if any such offerings are completed. They have
waived that right with respect to the common stock transactions described below.
If offerings totallingtotaling at least $6 million are not completed during the
6 months, the investor hasinvestors have the right to acquire up to 1,500a total of 1,126
additional shares of convertible preferred stock, the face value of which is
convertible at a pre-determinedthe option of the holders into common stock at $6.33 per share.
Any unconverted preferred stock is converted, at the applicable conversion
price, on April 13, 2002 in the case of the original stock and two years after
the first acquisition of any of the additional 1,5001,126 shares, if any are
acquired. The warrantwarrants expires on April 13, 2005.
AsOn August 3, 2000, we completed a result$4 million common stock financing
transaction with Millennium Partners, LP, or the Fund, an investment fund with
more than a billion dollars in assets under management. We received $3 million
of this transaction, the Companypurchase price at the closing and will receive the remaining $1 million
upon effectiveness of a registration statement covering the shares purchased by
the Fund. The Fund purchased our common stock at $4.33 per share. The Fund may
be entitled, pursuant to an adjustable warrant issued in connection with the
sale of common stock to the Fund, to receive additional shares of common stock
on eight dates beginning six months from the closing and every three months
thereafter. The number of additional shares the Fund may be entitled to on each
date will be based on the number of shares of common stock the Fund continues to
hold on each date and the market price of our common stock over a period prior
to each date. We will have the right, under certain circumstances, to cap the
number of additional shares by purchasing part of the entitlement from the Fund.
The Fund also received a warrant to purchase up to 101,587 shares of common
stock at $4.725 per share. This warrant is callable by us at $7.875 per
underlying share.
In addition, the Fund has adequate resourcesthe option for twelve months to fundpurchase up to
$3 million of additional common stock. On August 23, 2000 the Fund exercised
$1,000,000 of its operations intooption to purchase additional common stock at $5.53 per share.
The Fund paid $750,000 of the first quarterpurchase price in connection with the
27
closing on August 30, 2000, and will pay the remaining $250,000 upon
effectiveness of 2001.a registration statement covering the shares owned by the Fund.
At the closing on August 30, 2000, we issued to the Fund an adjustable warrant
similar to the one issued on August 3, 2000. This adjustable warrant was
canceled by agreement between us and the Fund on November 1, 2000. The Company hasFund also
received a warrant to purchase up to 19,900 shares of common stock at $6.03 per
share. This warrant is callable by us at $10.05 per underlying share.
We have limited liquidity and capital resources and must obtain significant
additional capital resources in the near future in order to sustain itsour product
development efforts. Substantial additional funds will be required to support
the Company'sour research and development programs, for acquisition of technologies and
intellectual property rights, for preclinical and clinical testing of itsour
anticipated products, pursuit of regulatory approvals, acquisition of capital
equipment, laboratory and office facilities, establishment of production
capabilities and for general and administrative expenses. The Company'sOur ability to obtain
additional capital will be substantially dependent on itsour ability to obtain
partnering support for itsour stem cell technology and, in the near term, on itsour
ability to realize proceeds from the sale, assignment or sublease of itsour
facilities in Rhode Island. There can be
no assurance that the Company will succeed in any or all of these efforts, and
failureFailure to do so will have a material adverse effect
on the Company's liquidity and capital resources. Until the Company'sour operations generate
significant revenues from product sales, the Companywe must rely on cash reserves and
proceeds from equity and debt offerings, proceeds from the transfer or sale of
itsour intellectual property rights, equipment or facilities, government grants and
funding from collaborative arrangements, if obtainable, to fund itsour operations.
The Company intendsWe intend to pursue opportunities to obtain additional financing in the
future through equity and debt financings, lease agreements related to
capital equipment, grants and collaborative research
arrangements. The source, timing and availability of any future financing will
depend principally upon market conditions, interest rates and, more
specifically, on the Company'sour progress in itsour exploratory, preclinical and future
clinical development programs. Lack of necessary funds may require the Companyus to delay,
reduce or eliminate some or all of itsour research and product development programs
or to license itsour potential products or technologies to third parties. No assurance can be given that
funding willFunding
may not be available when needed, if atneeded--at all, or on terms acceptable to the Company.
While the Company'sour cash requirements may vary, as noted above, the Companywe currently expectsexpect
that itsour existing capital resources, andincluding income earned on invested
capital, will be sufficient to fund itsour operations into the first quarter of
2001. This situationOur cash requirements may change,vary, however, depending on numerous factors.
Lack of necessary funds may require us to delay, scale back or eliminate some or
all of our research and product development programs and/or our capital
expenditures or to license our potential products or technologies to third
parties.
YEAR 2000
The Company tested its material software applications to determine whether
each program was prepared to accommodate date information for the year 2000 and
beyond, and found them to be year 2000 compliant. The Company also tested the
status of its facilities systems such as phones, voice mail, heating/ air
conditioning, electricity and security systems and its laboratory and
manufacturing equipment, and polled its major suppliers and vendors, to
determine if they are year 2000 compliant, again without identifying any
problems. Company has not to date encountered any significant year 2000
problems, but is continuing to monitor for potential issues. The costs of
testing and monitoring have been and are expected to continue to be immaterial
to the Company's operating results, but there can be no assurance that no
problem will reveal itself in the future, or that if a problem does occur it
will not have an adverse effect on the Company's operations or financial
results.
26
ITEM 7A. QUANTITIVEQUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK
As of the year ended December 31, 1999, the Company did not maintain any
investments that were exposed to market risk from changes in interest rates or
the fair market value of such investments. Interest
28
rate risk with respect to the CompanyCompany's short and long-term debt is considered
to be immaterial. As of the year ended December 31, 1999, the Company did not
maintain any hedge positions.
27Our investment in 126,193 shares of Modex Therapeutics Ltd. Stock was not
exposed to market risk as of December 31, 1999, however Modex shares were
offered in an IPO on the Swiss Neue Market on June 23, 2000 at a price of 168.00
Swiss francs. At June 30, 2000 our shares were valued at $19,220,165, based on
the per share price of $152.31 which we converted from the market price of
247.50 Swiss francs on June 30, 2000. The market price of the Modex stock on
October 31, 2000 was 329.50 Swiss francs, which converts to $183.28 using
exchange rates on that date, which represents an estimated fair market value of
$23,128,598 for our holdings. Our value in this investment is subject to both
equity price risk and foreign currency exchange risk. From the date of the
Modex IPO to the date hereof, the Modex closing share price has fluctuated from
a low of 200.00 Swiss francs on June 23, 2000 to a high of 390.00 Swiss francs
on October 6, 2000. If we were to seek to liquidate all or part of our
investment in Modex, our proceeds would depend on the share price and foreign
currency exchange rates at the time of conversion. Additionally, if we sell a
sizable portion of our holdings, we may have to sell these shares at a discount
to market price. We are restricted from any sale of our shares in Modex until
December 23, 2000.
The company's sole market risk sensitive instrument is:
MARKET VALUE EXPECTED
ASSOCIATED AT JUNE 30, FUTURE
NO. OF SHARES DESCRIPTION RISKS 2000 CASH FLOWS
123,193 Modex Equity/Foreign $19,220,165 (1)
Therapeutics Currency
Translation
- ------------------------
(1) Although the company has not formally adopted a liquidation plan for this
investment, liquidation may be necessary to meet operating cash flow
requirements. Under the agreement with Modex, the company is restricted from
selling its holding through December 23, 2000.
29
ITEM 8. FINANCIAL STATEMENTS AND SUPPLEMENTARY DATA
REPORT OF INDEPENDENT AUDITORS
Stockholders and Board of Directors
CytoTherapeutics, Inc.
We have audited the accompanying consolidated balance sheets of
CytoTherapeutics, Inc. as of December 31, 1999 and 1998, and the related
consolidated statements of operations, changes in redeemable common stock and
stockholders' equity and cash flows for each of the three years in the period
ended December 31, 1999. These financial statements are the responsibility of
the Company's management. Our responsibility is to express an opinion on these
financial statements based on our audits.
We conducted our audits in accordance with auditing standards. Those
standards require that we plan and perform the audit to obtain reasonable
assurance about whether the financial statements are free of material
misstatement. An audit includes examining, on a test basis, evidence supporting
the amounts and disclosures in the financial statements. An audit also includes
assessing the accounting principles used and significant estimates made by
management, as well as evaluating the overall financial statement presentation.
We believe that our audits provide a reasonable basis for our opinion.
In our opinion, the financial statements referred to above present fairly,
in all material respects, the consolidated financial position of
CytoTherapeutics, Inc. at December 31, 1999 and 1998, and the consolidated
results of its operations and its cash flows for each of the three years in the
period ended December 31, 1999, in conformity with accounting principles
generally accepted in the United States.
ERNST & YOUNG LLP
Providence, Rhode Island
April 14, 2000
2830
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
CONSOLIDATED BALANCE SHEETS
DECEMBER 31,
-----------------------------
1999 1998
------------- -------------
ASSETS
Current assets:
Cash and cash equivalents................................. $ 4,760,064 $ 7,864,788
Marketable securities..................................... -- 9,520,939
Accrued interest receivable............................... 42,212 206,609
Technology sale receivable................................ 3,000,000 --
Debt service fund......................................... 609,905 --
Other current assets...................................... 3,000,000 --558,674 841,674
------------- -------------
Total current assets........................................ 8,970,855 18,434,010
Property held for sale...................................... 3,203,491 --
Property, plant and equipment, net.......................... 5,251,3761,747,885 8,356,009
Other assets, net........................................... 1,858,768 6,075,663
------------- -------------
Total assets................................................ $ 16,080,99915,780,999 $ 32,865,682
============= =============
LIABILITIES AND STOCKHOLDERS' EQUITY
Current liabilities:
Accounts payable.......................................... $ 631,315 $ 710,622
Accrued expenses.......................................... 2,905,0682,605,068 1,020,119
Deferred revenue.......................................... -- 2,500,000
Current maturities of capitalized lease obligations....... 324,167 317,083
Current maturities of long-term debt...................... -- 1,000,000
------------- -------------
Total current liabilities................................... 3,860,5503,560,550 5,547,824
Capitalized lease obligations, less current maturities...... 2,937,083 3,261,667
Long-term debt, less current maturities..................... -- 500,000
Deposits.................................................... 26,000 --
Deferred Rent............................................... 502,353 222,673
Commitments and contingencies
Redeemable common stock, $.01 par value; 524,337 shares
issued and outstanding at December 31, 1999 and 1998...... 5,248,610 5,248,610
Common stock to be issued................................... -- 187,500
Stockholders' equity:
Convertible preferred stock, $.01 par value; 1,000,000
shares authorized; no shares issued and outstanding..... -- --
Common stock, $.01 par value; 45,000,000 shares
authorized; 18,635,565 and 17,800,323 shares issued and
outstanding at December 31, 1999 and 1998,
respectively............................................ 186,355 178,003
Additional paid-in capital................................ 123,917,758 122,861,606
Accumulated deficit....................................... (119,372,710) (103,664,084)
Unrealized losses on marketable securities................ -- (5,198)
------------- -------------
Accumulated othertotal comprehensive loss...................... (119,372,710) (103,669,282)
------------- -------------
Deferred compensation..................................... (1,225,000) (1,472,919)
------------- -------------
Total stockholders' equity.................................. 3,506,403 17,897,408
------------- -------------
Total liabilities and stockholders' equity.................. $ 16,080,99915,780,999 $ 32,865,682
============= =============
SEE ACCOMPANYING NOTES TO CONSOLIDATED FINANCIAL STATEMENTS.
2931
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
CONSOLIDATED STATEMENTS OF OPERATIONS
YEAR ENDED DECEMBER 31,
------------------------------------------
1999 1998 1997
------------ ------------ ------------
Revenue from collaborative agreements............... $ 5,021,707 $ 8,803,163 $ 10,617,443
Operating expenses:
Research and development.......................... 9,984,027 17,658,530 18,603,523
Acquired research and development................. -- -- 8,343,684
General and administrative........................ 4,927,303 4,602,758 6,158,410
Encapsulated Cell Therapy Windcell therapy wind down and Corporate
Relocation......................................corporate
relocation...................................... 6,047,806 -- --
------------ ------------ ------------
20,959,136 22,261,288 33,105,617
------------ ------------ ------------
Loss from operations................................ (15,937,429) (13,458,125) (22,488,174)
Other income (expense):
Interest income................................... 564,006 1,253,781 1,931,260
Interest expense.................................. (335,203) (472,400) (437,991)
Gain on partial sale of Modex..................... -- -- 3,386,808
Loss on sale/leaseback............................ -- -- (342,014)
Loss on equity investment......................... -- -- (105,931)
Other income (expense)............................ -- 48,914 (57,538)
------------ ------------ ------------
228,803 830,295 4,374,594
------------ ------------ ------------
Net loss............................................ $(15,708,626) $(12,627,830) $(18,113,580)
============ ============ ============
Basic and diluted net loss per share................ $ (.84) $ (.69) $ (1.08)
============ ============ ============
Shares used in computing basic and diluted net loss
per share......................................... 18,705,838 18,290,548 16,704,144
============ ============ ============
SEE ACCOMPANYING NOTES TO CONSOLIDATED FINANCIAL STATEMENTS.
3032
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
CONSOLIDATED STATEMENTS OF CHANGES IN REDEEMABLE COMMON STOCK AND STOCKHOLDERS'
EQUITY (CONTINUED)
REDEEMABLE
COMMON STOCK COMMON STOCK ADDITIONAL
---------------------- --------------------- PAID-IN ACCUMULATED
SHARES AMOUNT SHARES AMOUNT CAPITAL DEFICIT
------ ----------- ---------- -------- ------------ -------------
Balances, December 31, 1996............. 815,065 $ 8,158,798 15,614,333 $156,144 $107,649,659 $ (72,922,674)
Issuance of common stock................ -- -- 307,548 3,074 1,552,432 --
Issuance of common stock under the stock
purchase plan......................... -- -- 31,822 319 180,103 --
Deferred compensation recorded in
connection with the granting of stock
options............................... -- -- -- -- 1,750,000 --
Common stock issued pursuant to employee
benefit plan.......................... -- -- 25,588 256 169,196 --
Issuance of common stock--StemCells..... -- -- 1,219,381 12,194 7,381,206 --
Redeemable common stock lapses.......... (257,311) (2,575,688) 257,311 2,573 2,573,115 --
Exercise of stock options............... -- -- 75,237 752 244,427 --
Deferred compensation--amortization and
cancellations......................... -- -- (5,000) (50) (27,294) --
Change in unrealized losses on
marketable securities................. -- -- -- -- -- --
Change in cumulative translation
adjustment............................ -- -- -- -- -- --
Net loss................................ -- -- -- -- -- (18,113,580)
Comprehensive loss......................
-------- ----------- ---------- -------- ------------ -------------
Balances, December 31, 1997............. 557,754 $ 5,583,110 17,526,220 $175,262 $121,472,844 $ (91,036,254)
OTHER COMPREHENSIVE
INCOME
---------------------------
UNREALIZED
GAINS
(LOSSES) CUMULATIVE TOTAL
ON MARKETABLE TRANSLATION DEFERRED STOCKHOLDERS'
SECURITIES ADJUSTMENTS COMPENSATION EQUITY
------------- ----------- ------------- -------------
Balances, December 31, 1996............. $ 14,760 $(60,416) $ (90,118) $ 34,747,355
Issuance of common stock................ -- -- -- 1,555,506
Issuance of common stock under the stock
purchase plan......................... -- -- -- 180,422
Deferred compensation recorded in
connection with the granting of stock
options............................... -- -- (1,750,000) --
Common stock issued pursuant to employee
benefit plan.......................... -- -- -- 169,452
Issuance of common stock--StemCells..... -- -- -- 7,393,400
Redeemable common stock lapses.......... -- -- -- 2,575,688
Exercise of stock options............... -- -- -- 245,179
Deferred compensation--amortization and
cancellations......................... -- -- 137,298 109,954
Change in unrealized losses on
marketable securities................. (23,637) -- -- (23,637)
Change in cumulative translation
adjustment............................ -- 60,416 -- 60,416
Net loss................................ -- -- -- (18,113,580)
------------
Comprehensive loss...................... (18,076,081)
-------- -------- ----------- ------------
Balances, December 31, 1997............. $ (8,877) -- $(1,702,820) $ 28,900,155
SEE ACCOMPANYING NOTES TO CONSOLIDATED FINANCIAL STATEMENTS.
3133
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
CONSOLIDATED STATEMENTS OF CHANGES IN REDEEMABLE COMMON STOCK AND STOCKHOLDERS'
EQUITY (CONTINUED)
UNREALIZED
REDEEMABLE GAINS
COMMON STOCK COMMON STOCK ADDITIONAL (LOSSES)
---------------------- --------------------- PAID-IN ACCUMULATED ON MARKETABLE
SHARES AMOUNT SHARES AMOUNT CAPITAL DEFICIT SECURITIES
------ ----------- ---------- -------- ------------ ------------- -------------
Issuance of common stock........... -- -- -- -- -- -- --
Issuance of common stock under the
stock purchase plan.............. -- -- 43,542 $ 436 $ 83,622
Deferred compensation recorded in
connection with the granting of
stock options.................... -- -- -- -- -- -- --
Common stock issued pursuant to
employee benefit plan............ -- -- 84,812 848 143,025 -- --
Issuance of common
stock--StemCells................. -- -- 101,320 1,013 505,587 -- --
Redeemable common stock lapses..... (33,417) (334,500) 33,417 334 334,166 -- --
Exercise of stock options.......... -- -- 11,012 110 1,254 -- --
Deferred compensation--amortization
and cancellations................ -- -- -- -- 321,108 -- --
Change in unrealized losses on
marketable securities............ -- -- -- -- -- -- 3,679
Net loss........................... -- -- -- -- -- (12,627,830) --
Comprehensive loss.................
-------- ----------- ---------- -------- ------------ ------------- --------
Balances, December 31, 1998........ 524,337 $ 5,248,610 17,800,323 $178,003 $122,861,606 $(103,664,084) $ (5,198)
======== =========== ========== ======== ============ ============= ========
TOTAL
DEFERRED STOCKHOLDERS'
COMPENSATION EQUITY
------------- -------------
Issuance of common stock........... -- --
Issuance of common stock under the
stock purchase plan.............. $ 84,058
Deferred compensation recorded in
connection with the granting of
stock options.................... -- --
Common stock issued pursuant to
employee benefit plan............ -- 143,873
Issuance of common
stock--StemCells................. -- 506,600
Redeemable common stock lapses..... -- 334,500
Exercise of stock options.......... -- 1,364
Deferred compensation--amortization
and cancellations................ 229,901 551,009
Change in unrealized losses on
marketable securities............ -- 3,679
Net loss........................... -- (12,627,830)
------------
Comprehensive loss................. (12,624,151)
----------- ------------
Balances, December 31, 1998........ $(1,472,919) $ 17,897,408
=========== ============
3234
STEMCELLS, INC.
(FORMERLY, CYTOTHERAPEUTICS, INC.)
CONSOLIDATED STATEMENTS OF CHANGES IN REDEEMABLE COMMON STOCK AND STOCKHOLDERS'
EQUITY (CONTINUED)
UNREALIZED
REDEEMABLE GAINS
COMMON STOCK COMMON STOCK ADDITIONAL (LOSSES)
---------------------- --------------------- PAID-IN ACCUMULATED ON MARKETABLE
SHARES AMOUNT SHARES AMOUNT CAPITAL DEFICIT SECURITIES
------ ----------- ---------- -------- ------------ ------------- -------------
Issuance of common stock........... -- -- 196,213 $ 1,962 $ 318,221 -- --
Issuance of common stock under the
stock purchase plan.............. -- -- 57,398 574 41,619
Deferred compensation recorded in
connection with the granting of
stock options.................... -- -- -- -- -- -- --
Common stock issued pursuant to
employee benefit plan............ -- -- 90,798 908 102,502 -- --
Issuance of common
stock--StemCells................. -- -- -- -- -- -- --
Redeemable common stock lapses..... -- -- -- --
Exercise of stock options.......... -- -- 490,833 4,908 513,534 -- --
Deferred compensation--amortization
and cancellations................ -- -- -- -- 80,276 -- --
Change in unrealized losses on
marketable securities............ -- -- -- -- -- -- 5,198
Net loss........................... -- -- -- -- -- (15,708,626) --
Comprehensive loss.................
-------- ----------- ---------- -------- ------------ ------------- --------
Balances, December 31, 1999........ 524,337 $ 5,248,610 18,635,565 $186,355 $123,917,758 $(119,372,710) $ --
======== =========== ========== ======== ============ ============= ========
TOTAL
DEFERRED STOCKHOLDERS'
COMPENSATION EQUITY
------------- -------------
Issuance of common stock........... -- $ 320,183
Issuance of common stock under the
stock purchase plan.............. 42,193
Deferred compensation recorded in
connection with the granting of
stock options.................... -- --
Common stock issued pursuant to
employee benefit plan............ -- 103,410
Issuance of common
stock--StemCells.................
Redeemable common stock lapses.....
Exercise of stock options.......... -- 518,442
Deferred compensation--amortization
and cancellations................ 218,748247,919 328,195
Change in unrealized losses on
marketable securities............ -- 5,198
Net loss........................... -- (15,708,626)
------------
Comprehensive loss................. (15,703,428)
----------- ------------
Balances, December 31, 1999........ $(1,254,171)$(1,225,000) $ 3,506,403
=========== ============
SEE ACCOMPANYING NOTES TO CONSOLIDATED FINANCIAL STATEMENTS.
3335
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
CONSOLIDATED STATEMENTS OF CASH FLOWS
YEAR ENDED DECEMBER 31,
------------------------------------------
1999 1998 1997
------------ ------------ ------------
CASH FLOWS FROM OPERATING ACTIVITIES:
Net loss............................................loss.................................................... $(15,708,626) $(12,627,830) $(18,113,580)
Adjustments to reconcile net loss to net cash used for
operating activities:
Depreciation and amortization.....................amortization............................. 1,717,975 2,244,146 1,968,234
Acquired research and development.................development......................... -- 551,009 8,343,684
Amortization of deferred compensation.............compensation..................... 328,195 -- 109,954
Fair market adjustment for property held for sale......... 300,000 -- --
Other non-cash charges............................charges.................................... 320,183 410,173 105,931
Gain on investment................................investment........................................ -- -- (3,386,808)
Loss on sale of fixed assets......................assets.............................. 1,117,286 -- 413,856
Loss on sale of intangibles.......................intangibles............................... 440,486
Changes in operating assets and liabilities:
Accrued interest receivable.....................receivable............................. 164,397 346,577 100,004
Other current assets............................ 276,940assets.................................... 283,000 (265,665) (232,604)
Accounts payable and accrued expenses........... 1,644,142expenses................... 1,344,142 (2,378,613) (1,233,501)
Deferred rent...................................rent........................................... 279,680 -- --
Deferred revenue................................revenue........................................ (2,500,000) 2,483,856 (1,842,948)
------------ ------------ ------------
Net cash used in operating activities............... (12,489,342)activities....................... (11,913,282) (9,236,347) (13,767,778)
CASH FLOWS FROM INVESTING ACTIVITIES:
Proceeds from sale of Modex, net of cash disposed...disposed........... -- -- 2,958,199
Purchases of marketable securities..................securities.......................... (4,397,676) (18,982,387) (14,182,521)
Proceeds from sales of marketable securities........securities................ 13,923,813 22,573,625 23,736,242
Purchases of property, plant and equipment..........equipment.................. (192,747) (2,153,525) (7,710,126)
Proceeds on sale of fixed assets....................assets............................ 746,448 -- 8,003,926
Purchase of other investment........................investment................................ -- -- (250,000)
Acquisition of other assets......................... (552,251)assets................................. (558,311) (400,219) (1,599,418)
Disposal of other assets............................ 440,485assets.................................... 440,486 -- --
Acquisition of StemCells assets.....................assets............................. -- -- (640,490)
Advance to Cognetix.................................Cognetix......................................... -- -- (250,000)
Repayment from Cognetix.............................Cognetix..................................... -- -- 250,000
------------ ------------ ------------
Net cash provided by investing activities........... 9,968,073activities................... 9,962,013 1,037,494 10,315,812
CASH FLOWS FROM FINANCING ACTIVITIES:
Proceeds from issuance of redeemable common stock...stock........... -- -- --
Proceeds from issuance of common stock..............stock...................... 145,603 227,931 1,905,380
Proceeds from the exercise of stock options and warrants..........................................warrants.... 518,442 1,364 245,179
Proceeds from debt financings.......................financings............................... -- 1,259,300 --
Repayments of debt and lease obligations............obligations.................... (1,817,500) (1,366,655) (2,496,849)
------------ ------------ ------------
Net cash provided by (used in) financing activities........................................activities......... (1,153,455) 121,940 (346,290)
Effect of exchange rate changes on cash and cash
equivalents.......................................equivalents............................................... -- -- (181,627)
------------ ------------ ------------
Decrease in cash and cash equivalents...............equivalents....................... (3,104,724) (8,076,913) (3,979,883)
Cash and cash equivalents, January 1................1........................ 7,864,788 15,941,701 19,921,584
------------ ------------ ------------
Cash and cash equivalents, December 31..............31...................... $ 4,760,064 $ 7,864,788 $ 15,941,701
============ ============ ============
Supplemental disclosure of cash flow information:
Interest paid.....................................paid............................................. $ 335,203 $ 444,047 $ 436,461
Non-cash transaction:
In December 1999, the Company sold intellectual property related to its
encapsulated cell technology. In association with the transaction, the Company
recorded a receivable of $3,000,000 and reduced intangible assets.
SEE ACCOMPANYING NOTES TO CONSOLIDATED FINANCIAL STATEMENTS.
3436
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
DECEMBER 31, 1999
1. NATURE OF BUSINESS
StemCells, Inc. (formerly CytoTherapeutics, Inc.) (the "Company") is a
biopharmaceutical company engaged in the development of novel stem cell
therapies designed to treat human diseases and disorders.
2. SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES
PRINCIPLES OF CONSOLIDATION
The consolidated financial statements include accounts of the Company and
StemCells California, Inc., a wholly owned subsidiary. Significant intercompany
accounts have been eliminated in consolidation.
USE OF ESTIMATES
The preparation of the consolidated financial statements in conformity with
generally accepted accounting principles requires management to make estimates
and assumptions that affect the amounts reported in the consolidated financial
statements and accompanying notes. Actual results could differ from those
estimates.
CASH EQUIVALENTS AND MARKETABLE SECURITIES
Cash equivalents include funds held in investments with original maturities
of three months or less when purchased. The Company's policy regarding selection
of investments, pending their use, is to ensure safety, liquidity, and capital
preservationreservation while obtaining a reasonable rate of return. Marketable securities
consist of investments in agencies of the U.S. government, investment grade
corporate notes and money market funds. The fair values for marketable
securities are based on quoted market prices.
The Company determines the appropriate classification of cash equivalents
and marketable securities at the time of purchase and reevaluates such
designation as of each balance sheet date. The Company classifies such holdings
as available-for-sale securities, which are carried at fair value, with
unrealized gains and losses reported as a separate component of stockholders'
equity.
PROPERTY HELD FOR SALE
As a result of the Company's decision to exit the encapsulated cell
technology and relocate its corporate headquarters to Sunnyvale, CA, certain
property considered by management to no longer be necessary has been made
available for sale or lease. The aggregate carrying value of such property has
been reviewed by management, subject to appraisal and adjusted downward to
estimated market value.
PROPERTY, PLANT AND EQUIPMENT
Property, plant and equipment, including that held under capitalized lease
obligations, is stated at cost and depreciated using the straight-line method
over the estimated life of the respective asset, as follows:
BuildingBuildings and improvements 3--15improvements.................................. 3-15 years
Machinery and equipment 3--10equipment..................................... 3-10 years
Furniture and fixtures 3--10fixtures...................................... 3-10 years
37
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
2. SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (CONTINUED)
PATENT COSTS
The Company capitalizes certain patent costs related to patent applications.
Accumulated costs are amortized over the estimated economic life of the patents,
not to exceed 17 years, using the straight-line method, commencing at the time
the patent is issued. Costs related to patent applications are written off to
expense at the time such patents are deemed to have no continuing value. At
December 31, 1999 and 1998, total costs capitalized were $718,000 and $4,285,000
and the related accumulated amortization were $9,000
35
CYTOTHERAPEUTICS, INC.
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
2. SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (CONTINUED) and $347,000, respectively.
Patent expense totaled $539,000, $3,000, and $365,000 in 1999, 1998 and 1997,
respectively.
In December 1999, the Company sold its Encapsulated Cell Technology ("ECT")
to Neurotech, S.A. for an initial payment of $3,000,000, royalties on future
product sales, and a portion of certain Neurotech revenues from third parties,
in return for the assignment to Neurotech of intellectual property assets
relating to ECT. In addition, the Company retained certain non-exclusive rights
to use ECT in combination with its proprietary stem cell technology and in the
field of vaccines for prevention and treatment of infectious diseases. The
patent portfolio that was sold had a net book value of $3,180,000. The loss on
this transaction and expenses related to the write-down of ECT are included in
wind-down expenses on the Company's Consolidated Statement of Operations.
STOCK BASED COMPENSATION
The Company grants qualified stock options for a fixed number of shares to
employees with an exercise price equal to the fair market value of the shares at
the date of grant. The Company accounts for stock option grants in accordance
with APB Opinion No. 25, ACCOUNTING FOR STOCK ISSUED TO EMPLOYEES, and,
accordingly, recognizes no compensation expense for qualified stock option
grants.
For certain non-qualified stock options granted to non-employees, the
Company accounts for these grants in accordance with FAS No. 123--ACCOUNTING FOR
STOCK-BASED COMPENSATION AND EITF96-18--ACCOUNTING FOR EQUITY INSTRUMENTS THAT
ARE ISSUED TO OTHER THAN EMPLOYEES FOR ACQUIRING, OR IN CONJUNCTION WITH
SELLING, GOODS OR SERVICES, and accordingly, recognizes as compensation expenseconsulting expenses
the excess of the deemedestimated fair value of the common stock
issuable upon exercise of such options overas calculated using the aggregate exercise price of such
options.Black-Scholes
valuation model. Fair value is determined using methodologies allowable by FAS
No. 123. The compensationcost is amortized over the vesting period of each option or the
recipient's term of employment,contractual arrangement, if shorter.
INCOME TAXES
The liability method is used to account for income taxes. Deferred tax
assets and liabilities are determined based on differences between financial
reporting and income tax bases of assets and liabilities, as well as net
operating loss carry forwards, and are measured using the enacted tax rates and
rates under laws that are expected to be in effect when the differences reverse.
Deferred tax assets may be reduced by a valuation allowance to reflect the
uncertainty associated with their ultimate realization.
REVENUE FROM COLLABORATIVE AGREEMENTS
Revenues from collaborative agreements are recognized as earned upon either
the incurring of reimbursable expenses directly related to the particular
research plan or the achievement of certain
milestones.38
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
2. SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (CONTINUED)
development milestones as defined within the terms of the collaborative
agreement. Payments received in advance of research performed are designated as
deferred revenue. Recorded revenues are not refundable in the event research
efforts are considered unsuccessful.
RESEARCH AND DEVELOPMENT COSTS
The company expenses all research and development costs as incurred.
NET LOSS PER SHARE
Net loss per share is computed using the weighted average number of shares
of common stock outstanding. Common equivalent shares from stock options and
warrants are excluded, as their effect is antidilutive.
RECENTLY ISSUED ACCOUNTING PRONOUNCEMENTS
The Securities Exchange Commission's recently issued Staff Accounting
Bulletin No. 101 provides guidance on revenue recognition that may impact the
Company's future reporting relative to recognizing revenues received from
collaborative and similar agreements. 36
CYTOTHERAPEUTICS, INC.
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999The Company does not expect this guidance
to result in significant changes to its existing revenue recognition policy,
subject to the specific terms of each individual collaborative agreement.
3. SALE OF 6% CUMULATIVE CONVERTIBLE PREFERRED STOCK
On April 13, 2000, the Company completed arrangements to sell 1,500 shares
of 6% cumulative convertible preferred stock plus a warrant for 75,000 shares of
the Company's common stock to a membertwo members of its Board of Directors for
$1,500,000, on terms more favorable to the Company than it was then able to
obtain from outside investors. The shares are convertible at the option of the
holderholders into common stock at a
price to be determined by reference to$3.77 per share (based on the priceface value of the
Company's common stock
for a period approximately from April 12, 2000 through the twentieth trading day
following the filing of this Form 10-K.preferred shares). The conversion price may be below the trading market price of
the stock at the time of conversion. The holderCompany has valued the beneficial
conversion feature using the intrinsic value method reflecting the April 13,
2000 commitment date and the most beneficial per share discount available to the
preferred shareholders. Such value was $265,000 and will be treated as a deemed
dividend as of the commitment date. The holders of the preferred stock hashave
liquidation rights equal to histheir original investment plus accrued but unpaid
dividends. The investorinvestors would be entitled to make additional investments in the
Company on the same terms as those on which the Company completes offerings of
its securities with third parties within 6 months, if any such offerings are
completed. If offerings totallingtotaling at least $6 million are not completed during
the 6 months, the investor hasinvestors have the right to acquire up to 1,5001,126 additional
shares of convertible preferred stock at a pre-determined$6.33 per share. Any unconverted
preferred stock is converted (based on the face value of the preferred shares),
at the applicable conversion price, on April 13, 2002 in the case of the
original stock and two years after the first acquisition of any of the
additional 1,5001,126 shares, if any are acquired. The warrant expires on April 13,
2005.
4. WIND-DOWN39
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
4.WIND-DOWN OF ENCAPSULATED CELL TECHNOLOGY RESEARCH AND DEVELOPMENT PROGRAM
Until mid-1999, the Company engaged in research and development in
encapsulated cell therapy technology, including a pain control program funded by
AstraZeneca Group plc. The results from the 85-patient double-blind,
placebo-controlled trial of our encapsulated bovine cell implant for the
treatment of severe, chronic pain in cancer patients did not, however, meet the
criteria AstraZeneca had established for continuing trials for the therapy, and
in June 1999 AstraZeneca terminated the collaboration, as allowed under the
terms of the original collaborative agreement signed in 1995.
As a result of termination, management determined in July 1999 to
restructure its research operations to abandon all further encapsulated cell
technology research and concentrate its resources on the research and
development of its proprietary platform of stem cell technologies.
The Company wound down its research and manufacturing operations in Lincoln,
Rhode Island, and relocated its remaining research and development activities,
and its corporate headquarters, to the facilities of its wholly owned
subsidiary, StemCells California, Inc., in Sunnyvale, California, in
October 1999. The Company terminated legal, professional and consulting
contractual arrangements in support of ECT research. The Company had used these
legal, professional and consulting contractual arrangements to meet regulatory
requirements in support of its research work, to support contractual
arrangements with clinical sites, to provide assistance at clinical sites in
administering therapy and documenting activities, and to assist in compliance
with FDA and other regulations regarding its clinical trials. ECT related patent
law work was also terminated. The Company also engaged professional consultants
in connection with the determination to exit its ECT activities and restructure
is operations, which concluded with the exit from ECT activities and relocation
of its corporate headquarters to California. The Company reduced its workforce
by approximately 58 employees who had been focused on ECT programs and 10
administrative employees. As a result, the Company sold excess furniture and
equipment in December 1999 and is seeking to sublease the science and
administrative facility and to sell the pilot manufacturing facility.
Wind-down expenses totaled approximately $6,048,000 for the year ended
December 31, 1999; no such expenses were incurred in 1998 and 1997. These
expenses relate to the wind-down of the Company's encapsulated cell technology
research and development program and the Company's other Rhode Island
operations, and the transfer of the Company's corporate headquarters to
Sunnyvale, CaliforniaCalifornia.
40
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
4.WIND-DOWN OF ENCAPSULATED CELL TECHNOLOGY RESEARCH AND DEVELOPMENT PROGRAM
(CONTINUED)
A description of these expenses, including the amounts and an accrual for the
Company'speriods of
recognition, are as follows:
TOTAL
THIRD QUARTER FOURTH QUARTER WIND-DOWN
1999 1999 EXPENSE
------------- -------------- ----------
Employee severance costs............................... $1,554,000 $ -- $1,554,000
Impairment losses(1):
Fixed assets......................................... 800,000 -- 800,000
ECT patents.......................................... 260,000 -- 260,000
---------- ---------- ----------
1,060,000 -- 1,060,000
Rhode Island facilities carrying costs(2):
Corporate headquarters............................... 702,000 -- 702,000
Pilot manufacturing plant............................ 562,000 -- 562,000
---------- ---------- ----------
1,264,000 -- 1,264,000
Employee outplacement.................................. 200,000 -- 200,000
RIPSAT settlement(3)................................... -- 1,172,000 1,172,000
Loss on sale of assets(4):
Fixed assets......................................... -- 318,000 318,000
ECT patents.......................................... -- 180,000 180,000
---------- ---------- ----------
-- 498,000 498,000
Write-down of pilot plant(5)........................... -- 300,000 300,000
---------- ---------- ----------
$4,078,000 $1,970,000 $6,048,000
========== ========== ==========
- ------------------------
(1) Management's estimate of the fixed asset impairment was derived from
communications with an outside auction house. The patent impairment loss was
based on preliminary negotiations with parties interested in acquiring the
patents.
(2) Facilities carrying costs include the operating lease payments, utilities,
property taxes, insurance, maintenance, interest and other non-employee
related expenses necessary to maintaining these facilities through the
expected date of settlement of a 1989disposition (June 30, 2000).
(3) The Company originally received funding agreement withfrom the Rhode Island Partnership
for Science and Technology ("RIPSAT"(RIPSAT) for purposes of conducting ECT
activities conditioned upon maintaining the operating within the state.
RIPSAT claimed that the Company's decision to exit ECT activities and close
the Rhode Island operation was in violation of the funding arrangement and
that the Company was obligated to return a portion of the funding proceeds.
Although the Company disputed these claims, during the fourth quarter of
1999, management determined it was in the best interest of the company to
settle the issue.
(4) The Company held an auction to sell all ECT fixed assets. Proceeds from that
sale resulted in a loss, which was related to machinery and equipment
($292,000), and furniture and fixtures ($26,000).
41
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
associated
withNOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
4.WIND-DOWN OF ENCAPSULATED CELL TECHNOLOGY RESEARCH AND DEVELOPMENT PROGRAM
(CONTINUED)
(5) The write-down of the pilot plant was based on an independent property
appraisal, which was not available during the third quarter, when the
Company reached a decision to exit ECT activities and relocate the corporate
headquarters.
At December 31, 1999, the Company's $1.6 million wind-down reserve included
approximately $1.2 million for the RIPSAT settlement and approximately
$0.4 million for Rhode Island facility costs.
Property held for sale at December 31, 1999, consisted of $3.2 million
relating to the Company's pilot manufacturing facility.plant facility located in Lincoln, Rhode Island.
The Company suspended depreciation of these assets totalling approximately
$140,000 for the quarter ended December 31, 1999. The balance reflected the
$300,000 write-down included as part of the additional wind-down expenses
recognized during the fourth quarter, in accordance with Financial Accounting
Standards Board Statement 121, which requires that long-lived assets be reviewed
for impairment whenever events or circumstances indicate that the carrying value
of the asset may not be recoverable. There were no such assets at December 31,
1998.
5. STEMCELLS CALIFORNIA, INC.
In September 1997, a merger of a wholly owned subsidiary of the Companycompany and
StemCells California, Inc. was completed in the form of a purchase. Through the
merger, the Company acquired StemCells California, Inc. for a purchase price
totaling approximately $9,475,000, consisting of 1,320,691 shares of the
Company's common stock, valued at $6,600,000 and options and warrants for the
purchase of 259,296 common shares at nominal consideration, valued at
$7,900,000 in the aggregate,$1,300,000, the assumption of certain liabilities of $934,000 and transaction
costs of $641,000. Options and warrants were valued utilizing the intrinsic
method, and the resultant value approximated the value determined using the
Black-Scholes method. The purchase price was allocated, through abased upon an asset
valuation study using income approach methods, to license agreements valued at
$1,131,000 to be amortized over three years and acquired research and
development of $8,344,000, which was expensed. The acquired research and
development had not reached scientific feasibility and had no alternative future
uses. As part of the acquisition of StemCells, Richard M. Rose, M.D., became
President, Chief Executive Officer and director of the Company and Dr. Irving
Weissman became a director of the Company.
Upon consummation of the merger, the Company entered into consulting
arrangements with the principal scientific founders of StemCells: Dr. Irving
Weissman, Dr. Fred H. Gage and Dr. David Anderson. Additionally, in connection
with the merger, the Company was granted an option by the former shareholders of
StemCells to repurchase 500,000 of the Company's shares of Common Stock
exchanged for StemCells shares, upon the occurrence of certain events.
To attract and retain Drs. Rose, Weissman, Gage and Anderson, and to
expedite the progress of the Company's stem cell program, the Company awarded
these individuals options to acquire a total of 37
CYTOTHERAPEUTICS, INC.
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
5. STEMCELLS CALIFORNIA, INC. (CONTINUED)
approximately 1.6 million shares
of the Company's common stock, at an exercise price of $5.25 per share, the
quoted market price at the grant date;date. Under the original grants, approximately
100,000 of these options were exercisable immediately on the date of grant,
1,031,000 of these options would vest and become exercisable only upon the
achievement of specified milestones related to the Company's stem cell
development program and the remaining 469,000 options would vest over eight
years. The expense associated with the grants that vested immediately was
considered non-employee compensation and was
42
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
5. STEMCELLS CALIFORNIA, INC. (CONTINUED)
based on the fair value of the options granted. The expense was considered
immaterial. In connection with the 469,000 options issued to a non-employee,
Dr. Anderson, the Company recorded deferred compensation of $1,750,000, the fair
value of such options at the date of grant, which will be amortized over an
eight-year period. The fair value was determined using the Black-Scholes method
with the following inputs: volatility .594, expected life 8 years, dividend
yield 0.0%, risk free rate 5.98%. If the milestones specified relating to the
1,031,000 option grantgranted to non-employees, Drs. Weissman and Gage, are achieved,
at that time the Companycompany will record compensation expense for the excess of the quoted market
price of the common stock over the exercise price of $5.25 per share for 562,000
options and the fair market
value for 469,000 of such options determined using the Black-Scholes method. The Companycompany has
also designated a pool of 400,000 options to be granted to persons in a position
to make a significant contribution to the success of the stem cell program.
Stem cell research will beis conducted pursuant to the provisions of an agreement
between the Companycompany and Drs. Weissman and Gage providing for a two-year research
plan. If the goals of the research plan are accomplished, the Company has agreed
to fund continuing stem cell research. Increases in stem cells research funding
of not more than 25% a year will be funded by the Company as long as the goals
of the research plan are being met. However, the Company will retain the option
of (i) ceasing or reducing neuralbrain stem cell research even if all research plan
goals are met, but will be required to accelerate the vesting of all
still-achievable performance based stock options, and (ii) ceasing or reducing
non-neuralnon-brain stem cell research even if all plan goals are being met by affording
the scientific research founders the opportunity to continue development of the
non-neuralnon-brain stem cell research by licensing the technology related to such
research to the founders in exchange for a payment to the Company equal to all
prior Company funding for such research, plus royalty payments.
6. MODEX
In October 1997, the Company completed a series of transactions, which
resulted in the establishment of its previously 50%-owned Swiss subsidiary,
Modex Therapeutiques, S.A.Therapeutics, Ltd. (Modex), (Modex) as an independent company. In the
transactions, the Company reduced its ownership interest from 50% to
approximately 25% in exchange for $4 million cash and elimination of its prior
contingent obligation to contribute an additional Sfr 2.4 million (approximately
$1.7 million) to Modex in July 1998. In the transactions, all of the put and
call arrangements between the Company and other stockholders of Modex were
eliminated and the Company forgave $463,000 due from Modex to the Company. The
Company recorded a gain on the transactions of $3,387,000.
In April 1998, Modex completed an additional equity offering, in which the
Company did not participate. This resulted in a reduction in the Company's
ownership to less than 20% ownership; therefore, the Company accountsaccounted for this
investment under the cost method.method at December 31, 1999.
The pre-existing royalty-bearing Cross License Agreement between the Company
and Modex was assigned by the Company to Neurotech S.A., a privately held French
company, as part of the sale of the intellectual property assets related to the
Company's encapsulated cell therapy technology to Neurotech. Under the terms of
the sale to Neurotech, the Company will receive a portion of revenues Neurotech
receives from Modex under the Cross License Agreement.
3843
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
7. MARKETABLE SECURITIES
During 1999, the Company sold all of its remaining marketable equitable
securities. At December 31, 1999, all of the Company's available funds were held
in cash and cash equivalents. The following is a summary of available-for-sale
securities held at December 31, 1998:
DECEMBER 31, 1998
---------------------------------------------------
GROSS GROSS
UNREALIZED UNREALIZED ESTIMATED
COST GAINS LOSSES GAINS FAIR VALUE
----------- ---------- ---------- -----------
U.S. government securities..................... $ 1,500,994 $1,720 $ (504) $ 1,502,210
U.S. corporate securities...................... 9,225,095 3,244 (9,658) 9,218,681
----------- ------ -------- -----------
Total debt securities.......................... $10,726,089 $4,964 $(10,162) 10,720,891
=========== ====== ========
Debt securities included in cash and cash
equivalents.................................. (1,199,952)
===========
Debt securities included in marketable
securities................................... $ 9,520,939
===========
8. PROPERTY, PLANT AND EQUIPMENT
Property, plant and equipment consists of the following:
DECEMBER 31,
-----------------------
1999 1998
---------- ----------
Building and improvements............................ $5,666,987$ 665,890 $5,665,077
Machinery and equipment.............................. 3,144,1071,691,096 9,887,251
Furniture and fixtures............................... 219,260 869,831
---------- ----------
9,030,3542,576,286 16,422,159
Less accumulated depreciation and amortization....... 3,778,978828,401 8,066,150
---------- ----------
$5,251,376$1,747,885 $8,356,009
========== ==========
Depreciation and amortization expense was $1,436,000, $1,720,000, and
$1,778,000 for the years ending December 31, 1999, 1998 and 1997, respectively.
As part of the Company's restructuring of its operations, sale of its
encapsulated cell technology ("ECT"), and relocation of its corporate
headquarters to Sunnyvale, California, the Company identified fixed assets
associated with the ECT or otherwise no longer needed. In December of 1999, the
Company disposed of these excess fixed assets, realizing proceeds of
approximately $746,000. TheseAt the time of the sale, these assets had a net book
value of approximately $1,063,000 after a third quarter write-down of $800,000.$800,000,
which was based on management's estimate of expected sale proceeds. The third
quarter write-down and actual fourth quarter loss were included as wind-down
expenses.
Certain property, plant and equipment have been acquired under capitalized
lease obligations. These assets totaled $5,827,000 and $6,587,000, at
December 31, 1999 and 1998, respectively, with related accumulated amortization
of $2,747,000 and $2,860,000 at December 31, 1999 and 1998, respectively. 39As a
44
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
8. PROPERTY, PLANT AND EQUIPMENT (CONTINUED)
result of the Company's decision to exit ECT and relocate to Sunnyvale, CA, this
property has been classified as held for sale at December 31, 1999.
9. OTHER ASSETS
Other assets are as follows:
DECEMBER 31,
-----------------------
1999 1998
---------- ----------
Patents, net......................................... $ 708,823 $3,938,755
License agreements, net.............................. 282,750 659,750
Security deposit--building lease..................... 750,000 750,000
Restricted cash...................................... -- 603,467
Deferred financing costs, net........................ 117,195 123,701
---------- ----------
$1,858,768 $6,075,663
========== ==========
The decrease in patents from 1999 to 1998 was primarily due to management's
decision to exit encapsulated cell technology and dispose of the related
intellectual property. Management reached this decision during the third quarter
of 1999, and established a reserve that included $260,000 directly related to
the write-down of encapsulated cell technology patents. During the fourth
quarter, management established an additional reserve that included a $180,000
loss associated with the sale of encapsulated cell technology patents worth
$3,180,000.
At December 31, 1999 and 1998, accumulated amortization was $857,000 and
$818,000, respectively, for patents and license agreements.
10. ACCRUED EXPENSES
Accrued expenses are as follows:
DECEMBER 31,
-----------------------
1999 1998
---------- ----------
Wind-down expenses................................... $1,634,522 $ --
External services.................................... $2,031,961 $97,439 412,253
Employee compensation................................ 306,342 262,679
Collaborative research............................... 222,140 196,505
Other................................................ 344,625 148,682
---------- ----------
$2,905,068 $1,020,119
========== ==========
The reserve for wind-down expenses included approximately $1,172,000
relating to the RIPSAT settlement (Notes 4 and 11) and approximately $463,000
for the estimated six months of lease payments and operating costs for the Rhode
Island Facilities through an expected disposal date of June 30, 2000.
45
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
11. LEASES
The Company has undertaken direct financing transactions with the State of
Rhode Island and received proceeds from the issuance of industrial revenue bonds
totaling $5,000,000 to finance the construction of its pilot manufacturing
facility. The related leases are structured such that lease payments will fully
fund all semiannual interest payments and annual principal payments through
maturity in August 2014. Fixed interest rates vary with the respective bonds'
maturities, ranging from 5.1% to 9.5%. The bonds contain certain restrictive
covenants which limit, among other things, the payment of cash dividends and the
sale of the related assets. In addition, the Company was required to maintain a
debt service reserve until December 1999. On March 3, 2000 the Company entered
into a settlement agreement with RIPSAT, the Rhode Island Industrial
Recreational Building Authority ("IRBA") and the Rhode Island Industrial
Facilities Corporation ("RIIFC"). The Company agreed to pay RIPSAT $1,172,000 in
full satisfaction of all obligations of the Company to RIPSAT under the Funding
Agreement dated as of June 22, 1989. On execution and delivery of this
Agreement, IRBA agreed to return to the Company the full amount of the Company's
debt serveservice reserve ("Reserve Funds"), approximately $610,000 of principal and
interest, relating to the bonds the Company has with IRBA and RIIFC. Such amount
has been classified as debt service funds in current assets of the consolidated
balance sheet. In order to avoid the loss of
40
CYTOTHERAPEUTICS, INC.
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
11. LEASES (CONTINUED) interest on the Reserve Funds due
to early termination of certain investments, the parties agreed that the Company
would render a net payment to RIPSAT in the amount of approximately $562,000.
In 1997, the Company completed construction of a new headquarters and
laboratory facility. In November 1997, the Company entered into sale and
leaseback agreements with a real estate investment trust. Under the terms of
these agreements, the Company sold its new facility for $8,000,000, incurring a
$342,000 loss on the sale. The Company simultaneously entered into a
fifteen-year lease for the facility. The lease agreement calls for minimum rent
of $750,000 for the first five years, $937,500 for years six to ten, $1,171,900
for years eleven to fourteen and $1,465,000 in year fifteen, with a $750,000
security deposit held for the term of the lease. The Company is recognizing rent
expense on a straight line basis. At December 31, 1999, the Company has incurred
$426,790 in deferred rent expense.
Future minimum capitalized lease obligations with non-cancelable terms in
excess of one year at December 31, 1999, are as follows:
2000........................................................ $ 606,268
2001........................................................ 589,217
2002........................................................ 519,719
2003........................................................ 436,909
2004........................................................ 425,713
Thereafter.................................................. 2,577,826
----------
Total minimum lease payments................................ 5,302,407
Less amounts representing interest.......................... 2,041,157
----------
Present value of minimum lease payments..................... 3,261,250
Less current maturities..................................... 324,167
----------
Capitalized lease obligations, less current maturities...... $2,937,083
==========
46
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
11. LEASES (CONTINUED)
Rent expense for the years ended December 31, 1999, 1998 and 1997, was
$947,000, $1,052,000 and $499,000, respectively.
12. LONG-TERM DEBT
Long-term debt is as follows:
DECEMBER 31,
-----------------------
1999 1998
---------- ----------
Term note payable, interest at the prime rate plus
1/2% (8.75% at December 31, 1998), principal
payments commence in August 1998, due ratably
through May 2000; secured by certain equipment
(prepaid during 1999).............................. $ -- $1,500,000
Current maturities of long-term debt................. -- 1,000,000
---------- ----------
Long-term debt, less current maturities.............. -- $ $500,000
========== ==========
41
CYTOTHERAPEUTICS, INC.
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
13. REDEEMABLE COMMON STOCK
In November 1996, the Company signed certain collaborative development and
licensing agreements with Genentech, Inc, including one under which Genentech
purchased 829,171 shares of redeemable common stock for $8.3 million to fund
development of products to treat Parkinson's disease. The Agreement also
provided that Genentech had the right, at its discretion, to terminate the
Parkinson's program at specified milestones in the program, and that if the
program were terminated, Genentech had the right to require the Company to
repurchase from Genentech the shares of the Company's common stock having a
value equal to the amount by which the $8.3 million exceeded the expenses
incurred by the Company in connection with such studiesprogram by more than
$1 million, based upon the share price paid by Genentech. Accordingly, the
common stock is classified as redeemable common stock until such time as the
related funds are expended. At December 31, 1998, $3,051,000 had been spent on
the collaboration with Genentech and, accordingly, the Company has reclassified
those common shares and related value to stockholders' equity. On May 21, 1998,
Genentech exercised its right to terminate the collaboration and negotiations
ensued with respect to the amount of redeemable common stock to be redeemed in
accordance with the agreement and the method of such redemption. In March 2000,
the Company reached a settlement of this matter with Genentech. Under the
settlement agreement, Genentech released the Company from any obligation to
redeem any shares of the Company's Common Stock held by Genentech. Accordingly,
the Company will reclassify the amount currently recorded as Redeemable Common
Stock ($5,248,000) to Stockholders' Equity in March 2000. The Company and
Genentech also agreed that all of the agreements between them were terminated
and that neither had any claim to the intellectual property of the other.
47
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
14. COMMON STOCK TO BE ISSUED
In 1998, the Company entered into an agreement with a Company advisor, under
which the advisor prepared a strategic and business overview and provided
related implementation support for the Company. The advisor agreed to accept
cash and the Company's common stock as partial payment for its services. In
1999, the Company issued the $187,500 of common stock due to the advisor.
15. STOCKHOLDERS' EQUITY
STOCK OPTION AND EMPLOYEE STOCK PURCHASE PLANS
The Company has adopted several stock plans that provide for the issuance of
incentive and nonqualified stock options, performance awards and stock
appreciation rights, at prices to be determined by the Board of Directors, as
well as the purchase of Common Stock under an employee stock purchase plan at a
discount to the market price. In the case of incentive stock options, such price
will not be less than the fair market value on the date of grant. Options
generally vest ratably over four years and are exercisable for ten years from
the date of grant or within three months of termination. At December 31, 1999,
the Company had reserved 2,603,736 shares of common stock for the exercise of
stock options.
42
CYTOTHERAPEUTICS, INC.
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
15. STOCKHOLDERS' EQUITY (CONTINUED)
The following table presents the combined activity of the Company's stock
option plans (exclusive of the plans noted below) for the years ended
December 31:
1999 1998 1997
-------------------------- --------------------------- --------------------------
WEIGHTED WEIGHTED WEIGHTED
AVERAGE AVERAGE AVERAGE
OPTIONS EXERCISE PRICE OPTIONS EXERCISE PRICE OPTIONS EXERCISE PRICE
--------- -------------- ---------- -------------- --------- --------------
Outstanding at January 1...... 1,654,126 $3.62 2,446,573 $7.48 2,423,025 $8.34
Granted....................... 536,078 1.08 1,174,118 1.70 679,074 5.33
Exercised..................... (604,362) 1.50 (11,012) .12 (82,737) 2.96
Canceled...................... (646,507) 5.31 (1,955,553) 7.08 (572,789) 9.21
--------- ----- ---------- ----- --------- -----
Outstanding at December 31.... 939,335 $2.65 1,654,126 $3.62 2,446,573 $7.48
========= ===== ========== ===== ========= =====
Options exercisable at
December 31................. 594,216 $3.44 1,108,936 $4.33 1,338,163 $7.79
========= ===== ========== ===== ========= =====
On July 10, 1998, the Company re-priced 751,018 outstanding stock options.
No compensation expense was recorded since the re-priced options carried an
exercise price equal to the market price of the Company's common stock on the
date of the re-pricing.
In addition to the options noted above, in conjunction with the StemCells
California merger, StemCells California options originally issued under a prior
StemCells California options plan were exchanged for options to purchase 250,344
shares of the Company's common stock at $.01 per share; 75,384 of these options
are exercisable at December 31, 1997, 96,750 of these options vest and become
exercisable only upon achievement of specified milestones, and the remaining
78,210 options vest over three years from the date of grant. The value of such
options utilizing the intrinsic method, which approximated the value determined
using the Black-Scholes method, was accounted for as part of the StemCells
California acquisition price. Additionally, the Company adopted the 1997
CytoTherapeutics, Inc. StemCells California Research Stock Option Plan (the
StemCells California Research Plan)
48
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
15. STOCKHOLDERS' EQUITY (CONTINUED)
whereby an additional 2,000,000 shares of Common Stock have been reserved.
During 1997, the Company awarded options under the StemCells Research Plan to
purchase 1.6 million shares of the Company's common stock to the Chief Executive
Officer and scientific founders of StemCells at an exercise price of $5.25 per
share;share. Under the original grants, approximately 100,000 of these options arewere
exercisable immediately on the date of grant, 1,031,000 of these options would
vest and become exercisable only upon achievement of specified milestones and
the remaining 469,000 options would vest over eight years. Options granted to
Dr. Rose, in his capacity as Chief Executive Officer, were valued using the
intrinsic value method, in accordance with the provisions of APB 25, ACCOUNTING
FOR STOCK ISSUED TO EMPLOYEES. Options granted to non-employees Drs. Weissman,
Gage and Anderson were accounted for using the fair value method in accordance
with the provisions of Statement of Financial Accounting Standards No. 123,
ACCOUNTING FOR STOCK-BASED COMPENSATION.
FAS 123 DISCLOSURES
The Company has adopted the disclosure provisions only of Statement of
Financial Accounting Standards No. 123, ACCOUNTING FOR STOCK-BASED COMPENSATION
("FAS 123") and accounts for its stock option plans in accordance with the
provisions of APB 25, ACCOUNTING FOR STOCK ISSUED TO EMPLOYEES.
43
CYTOTHERAPEUTICS, INC.
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
15. STOCKHOLDERS' EQUITY (CONTINUED)
The following table presents weighted average price and life information
about significant option groups outstanding at December 31, 1999:
OPTIONS OUTSTANDING OPTIONS EXERCISABLE
------------------------------------ ----------------------
WEIGHTED
AVERAGE WEIGHTED WEIGHTED
REMAINING AVERAGE AVERAGE
RANGE OF NUMBER CONTRACTUAL EXERCISE NUMBER EXERCISE
EXERCISE PRICES OUTSTANDING LIFE (YRS.) PRICE EXERCISABLE PRICE
- ------------------------------------------------ ----------- ----------- -------- ----------- --------
Less than $5.00................................. 755,398 8.50 $1.12 411,945 $ 1.02
$5.01--$10.00................................... 90,687 4.56 6.55 89,021 6.55
Greater than $10.00............................. 93,250 2.54 11.18 93,250 11.18
------- -------
939,335 594,216
======= =======
Pursuant to the requirements of FAS 123, the following are the pro forma net
loss and net loss per share amounts for 1999, 1998, and 1997, as if the
compensation cost for the option plans and the stock purchase plan had been
determined based on the fair value at the grant date for grants in 1999, 1998,
and 1997, consistent with the provisions of FAS 123:
1999 1998 1997
--------------------------- --------------------------- ---------------------------
AS REPORTED PRO FORMA AS REPORTED PRO FORMA AS REPORTED PRO FORMA
------------ ------------ ------------ ------------ ------------ ------------
Net loss............. $(15,708,626) $(15,764,569) $(12,627,830) $(14,919,389) $(18,113,580) $(19,924,437)
Net loss per share... $(.84) $(.84) $(.69) $(.82) $(1.08) $(1.19)
The weighted average fair value per share of options granted during 1999,
1998 and 1997 was $.88, $.82 and $3.40, respectively. The fair value of options
and shares issued pursuant to the stock purchase
49
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
15. STOCKHOLDERS' EQUITY (CONTINUED)
plan at the date of grant were estimated using the Black-Scholes model with the
following weighted average assumptions:
OPTIONS STOCK PURCHASE PLAN
------------------------------------ ------------------------------------
1999 1998 1997 1999 1998 1997
-------- -------- -------- -------- -------- --------
Expected life (years)........................ 5 5 5 5 .5 .5
Interest rate................................ 5.5% 5.2% 6.2% 5.0% 4.64% 5.5%
Volatility................................... 96.7% 63.5% 59.0% 96.7% 63.5% 59.0%
The Company has never declared nor paid dividends on any of its capital
stock and does not expect to do so in the foreseeable future.
The effects on 1999, 1998 and 1997 pro forma net loss and net loss per share
of expensing the estimated fair value of stock options and shares issued
pursuant to the stock purchase plan are not necessarily representative of the
effects on reporting the results of operations for future years as the period
presented includes only four, three or two years, respectively, of option grants
under the Company's plans. As required by FAS 123, the Company has used the
Black-Scholes model for option valuation, which method may not accurately value
the options described.
44
CYTOTHERAPEUTICS, INC.
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
15. STOCKHOLDERS' EQUITY (CONTINUED)
STOCK WARRANTS
In conjunction with StemCells California merger, the Company exchanged
StemCells California warrants for warrants to purchase 8,952 shares of Company
common stock at $4.71 per share.share; such warrants were valued using the intrinsic
value method which approximated the value determined using the Black-Scholes
method, and were accounted for as part of the purchase price. In conjunction
with various equipment leasing agreements, the Company hashad outstanding warrants
to purchase 31,545 shares of common stock at prices ranging from $4.00 to $9.00
per share. The warrants expire throughexpired in October 2000.
In connection with a public offering of common stock in April 1995, the
Company issued warrants to purchase 434,500 shares of common stock at $8 per
share. The warrants are nontransferable and expireexpired in April 2000, subject to
certain required exercise provisions. In addition to the foregoing rights, the
holder of such warrants has the right, in the event the Company issues
additional shares of common stock or other securities convertible into common
stock, to purchase at the then market price of such common stock, sufficient
additional shares of common stock to maintain the warrant holder's percentage
ownership of the Company's common stock at 15%. This right, subject to certain
conditions and limitations, expires in April 2000.
COMMON STOCK RESERVED
The Company has reserved 6,461,846 shares of common stock for the exercise
of options, warrants and other contingent issuances of common stock.
50
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
16. RESEARCH AGREEMENTS
In November 1997, StemCells California, Inc., a wholly owned subsidiary of
the Company, signed a Research Funding and Option Agreement with The Scripps
Research Institute ("Scripps") relating to certain stem cell research. Under the
terms of the Agreement, StemCells agreed to fund research in the total amount of
approximately $931,000 at Scripps over a period of three years. StemCells paid
Scripps approximately $77,000 in 1997, $307,000 in 1998, and $309,000 in 1999.
In addition, the Company agreed to issue to Scripps 4,837 shares of the
Company's common stock and a stock option to purchase 9,674 shares of the
Company's Common Stock with an exercise price of $.01 per share upon the
achievement of specified milestones. Under the Agreement, StemCells has an
option for an exclusive license to the inventions resulting from the sponsored
research, subject to the payment of royalties and certain other amounts, and is
obligated to make payments totaling $425,000 for achievement of certain
milestones.
In April 1997, the Company entered into an agreement with
Neurospheres, Ltd., which superseded all previous licensing agreements and
settled a dispute with Neurospheres. Under the terms of the settlement, the
Company has an exclusive royalty bearing license for growth-factor responsive
stem cells for transplantation. Neurospheres had an option to acquire
co-exclusive rights but did not exercise by the April 1998 deadline. The Company
retains exclusive rights for transplantation. The parties have no further
research obligations to each other.other, and the Company is under no obligation to
provide additional funding.
In February 1997, CytoTherapeutics and Cognetix, Inc. entered into a
Collaboration and Development Agreement related to the Company's former
encapsulated cell technology. As part of the agreement with Cognetix, the
Company purchased $250,000 of Cognetix preferred stock and, subject to certain
milestones, was obligated to purchase as much as $1,500,000 of additional
Cognetix stock over the next 45
CYTOTHERAPEUTICS, INC.
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
16. RESEARCH AGREEMENTS (CONTINUED)
year. In July 1997, the Company loaned $250,000 to
Cognetix which was repaid with interest in October 1997. In October 1998, the
Company sold the $250,000 of preferred stock back to Cognetix for $298,914. The
Company is under no obligation to provide additional funding under the
agreement.
In 1996, the Company signed certain collaborative development and licensing
agreements with Genentech, Inc. Under the terms of one of those agreements,
Genentech purchased 829,171 shares of redeemable common stock for $8.3 million
to fund development of products to treat Parkinson's disease. Genentech had the
right, at its discretion, to terminate the Parkinson's program at specified
milestones in the program. The Agreement also provided that if the Parkinson's
program were terminated and the funds of the Company received from the sale of
stock to Genentech pursuant to the Parkinson's agreement exceeded the expenses
incurred by the Company in connection with such studiesprogram by more than
$1 million, Genentech had the right to require the Company to repurchase from
Genentech shares of the Company's common stock having a value equal to the over
funding, based upon the share price paid by Genentech. As such, the common stock
purchased by Genentech has been classified as redeemable common stock until the
funds are expended on the program. On May 21, 1998, Genentech exercised its
right to terminate the collaboration and negotiations ensued with respect to the
amount of redeemable common stock to be redeemed in accordance with the
agreement and the method of such redemption. In March 2000 the Company announced
the settlement of this matter with Genentech. (SEE NOTE 18--"SUBSEQUENT EVENTS"
RELATING TO THE SETTLEMENT OF AND TERMINATION OF THE GENENTECH AGREEMENTS.)Genentech and at that time the redeemable
common stock was reclassified to common stock. The Company is under no
obligation to provide additional funding to Genentech, Inc.
In March 1995, the Company signed a collaborative research and development
agreement with AstraZeneca for the development and marketing of certain
encapsulated-cell products to treat pain.
51
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
16. RESEARCH AGREEMENTS (CONTINUED)
AstraZeneca made an initial, nonrefundable payment of $5,000,000, included in
revenue from collaborative agreements in 1995, a milestone payment of $3,000,000
in 1997 and was to remit up to an additional $13,000,000 subject to achievement
of certain development milestones. Under the agreement, the Company was
obligated to conduct certain research and development pursuant to a four-year
research plan agreed upon by the parties. Over the term of the research plan,
the Company originally expected to receive annual payments of $5 million to
$7 million from AstraZeneca, which was to approximate the research and
development costs incurred by the Company under the plan. Subject to the
successful development of such products and obtaining necessary regulatory
approvals, AstraZeneca was obligated to conduct all clinical trials of products
arising from the collaboration and to seek approval for their sale and use.
AstraZeneca had the exclusive worldwide right to market products covered by the
agreement. Until the later of either the expiration of all patents included in
the licensed technology or a specified fixed term, the Company was entitled to a
royalty on the worldwide net sales of such products in return for the marketing
license granted to AstraZeneca and the Company's obligation to manufacture and
supply products. AstraZeneca had the right to terminate the original agreement
beginning April 1, 1998. On June 24, 1999, AstraZeneca informed the Company of
the results of AstraZeneca's analysis of the double-blind, placebo-controlled
trial of the Company's encapsulated bovine cell implant for the treatment of
severe, chronic pain in cancer patients. AstraZeneca determined that, based on
criteria it established, the results from the 85-patient trial did not meet the
minimum statistical significance for efficacy established as a basis for
continuing worldwide trials for the therapy. AstraZeneca therefore indicated
that it did not intend to further develop the bovine cell-containing implant
therapy and executed its right to terminate the agreement. The Company has no
additional funding obligations with AstraZeneca.
The Company has entered into other collaborative research agreements whereby
the Company funds specific research programs. Pursuant to such agreements, the
Company is typically granted rights to the related intellectual property or an
option to obtain such rights on terms to be agreed, in exchange for
46
CYTOTHERAPEUTICS, INC.
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
16. RESEARCH AGREEMENTS (CONTINUED) research
funding and specified royalties on any resulting product revenue. The Company's
principal academic collaborations had been with Brown University and
Dr. Aebischer and Centre Hospitalier Universitaire Vaudois in Switzerland.
However, with the termination of the Company's Encapsulated Cell Technology
program and its focusing on the stem cell field, its principal academic
collaborations are now with the Scripps Institute and the Oregon Health Science
University. Research and development expenses incurred under these
collaborations amounted to approximately $868,000, $1,259,000, and $1,326,000
for the years ended December 31, 1999, 1998 and 1997, respectively. The Company
has no other significant collaborative research funding obligations.
17. INCOME TAXES
Due to net losses incurred by the Company in each year since inception, no
provision for income taxes has been recorded. At December 31, 1999, the Company
had tax net operating loss carry forwards of $96,195,000 and research and
development tax credit carry forwards of $4,035,000 which expire at various
times through 2019. Due to the "change in ownership" provisions of the Tax
Reform Act of 1986, the Company's utilization of its net operating loss carry
forwards and tax credits may be subject to annual limitation in future periods.
52
STEMCELLS, INC.
(FORMERLY CYTOTHERAPEUTICS, INC.)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
17. INCOME TAXES (CONTINUED)
Significant components of the Company's deferred tax assets and liabilities
are as follows:
DECEMBER 31,
---------------------------
1999 1998
------------ ------------
Deferred tax assets:
Capitalized research and development costs................ $ 4,331,000 $ 28,124,000
Net operating losses...................................... 38,478,000 10,786,000
Research and development credits.......................... 4,035,000 3,646,000
Other..................................................... 928,000 235,000
------------ ------------
47,772,000 42,791,000
Deferred tax liabilities:
Patents................................................... (246,000) (1,537,000)
------------ ------------
47,526,000 41,254,000
Valuation allowance....................................... (47,526,000) (41,254,000)
------------ ------------
Net deferred tax assets..................................... $ -- $ --
============ ============
Since there is uncertainty relating to the ultimate use of the loss carry
forwards and tax credits, a valuation allowance has been recognized at
December 31, 1999 and 1998, to fully offset the Company's deferred tax assets.
The valuation allowance increased $6,272,000 in 1999, due primarily to the
increases in net operating loss carry forwards and tax credits offset by
reduction in capitalized research and development costs .
18. EMPLOYEE RETIREMENT PLAN
The Company has a qualified defined contribution plan covering substantially
all employees. Participants are allowed to contribute a fixed percentage of
their annual compensation to the plan and the Company may match a percentage of
that contribution. The Company matches 50% of employee 47
CYTOTHERAPEUTICS, INC.
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (CONTINUED)
DECEMBER 31, 1999
18. EMPLOYEE RETIREMENT PLAN (CONTINUED)
contributions, up to 6%
of employee compensation, with the Company's common stock. The related expense
was $103,000, $146,000, and $169,000 for the years ended December 31, 1999, 1998
and 1997, respectively.
19. CONTINGENCIES
The Company is routinely involved in arbitration, litigation and other
matters as part of the ordinary course of its business. While the resolution of
any matter may have an impact on the Company's financial results for a
particular reporting period, management believes the ultimate disposition of
these matters will not have a materially adverse effect on the Company's
consolidated financial position or results of operations.
20. SUBSEQUENT EVENTS
On April 13, 2000, the Company completed arrangements to sell 1,500 shares
of 6% cumulative convertible preferred stock plus a warrant for 75,000 shares of
the Company's common stock to a member of its Board of Directors for $1,500,000,
on terms more favorable than it was then able to obtain from outside investors.
(SEE NOTE 3--"SALE OF 6% CUMULATIVE CONVERTIBLE PREFERRED STOCK.")
4853
ITEM 9. CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND
FINANCIAL DISCLOSURE
None.
PART III
ITEM 10. DIRECTORS AND EXECUTIVE OFFICERS OF THE REGISTRANT, PROMOTERS AND
CONTROL
DIRECTORS AND EXECUTIVE OFFICERS
The sections entitled "Election of Directors" and "Executive Officer" in the
Company's definitive proxy statement for its 2000 Annual Meeting of Shareholders
are hereby incorporated by reference.
ITEM 11. EXECUTIVE COMPENSATION
The section entitled "Executive Compensation" in the Company's definitive
proxy statement for its 2000 Annual Meeting of Shareholders is hereby
incorporated by reference.
ITEM 12. SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT
The section entitled "Share Ownership" in the Company's definitive proxy
statement for its 2000 Annual Meeting of Shareholders is hereby incorporated by
reference.
ITEM 13. CERTAIN RELATIONSHIPS AND RELATED TRANSACTIONS
The section entitled "Certain Relationships and Related Transactions" in the
Company's definitive proxy statement for its 2000 Annual Meeting of Shareholders
is hereby incorporated by reference.
PART IV
ITEM 14. EXHIBITS, FINANCIAL STATEMENT SCHEDULES AND REPORTS ON FORM 8-K
(a) Documents filed as part of this Form 10-K.
(1) Financial Statement Schedules:
Schedules not included herein are omitted because they are not applicable or
the required information appears in the Financial Statements or Notes thereto.
(2) Exhibits.
EXHIBIT NO. TITLE OR DESCRIPTION
----------- --------------------
3.1* Restated Certificate of Incorporation of the Registrant.
3.2++ Amended and Restated By-Laws of the Registrant.
4.1* Specimen Common Stock Certificate.
4.2++++ Form of Warrant Certificate issued to a certain purchaser of
the Registrant's Common Stock in April 1995.
10.4* Amendment to Registration Rights dated as of February 14,
1992 among the Registrant and certain of its stockholders.
10.15* Form of at-will Employment Agreement between the Registrant
and most of its employees.
10.20* Form of Agreement for Consulting Services between the
Registrant and members of its Scientific Advisory Board.
10.21* Form of Nondisclosure Agreement between the Registrant and
its Contractors.
4954
EXHIBIT NO. TITLE OR DESCRIPTION
----------- --------------------
10.28* Master Lease and Warrant Agreement dated April 23, 1991
between the Registrant and PacifiCorp Credit, Inc.
10.29* 1988 Stock Option Plan.
10.30* 1992 Equity Incentive Plan.
10.31* 1992 Stock Option Plan for Non-Employee Directors.
10.32* 1992 Employee Stock Purchase Plan.
10.41**!!!! Development and Supply Agreement dated December 1993 between
Registrant and AKZO Faser AG.
10.43##** Research Agreement dated as of February 1, 1994 between
Genentech, Inc. and Registrant.
10.44##** Research Agreement dated as of March 16, 1994 between
NeuroSpheres, Ltd. and Registrant.
10.47++ Term Loan Agreement dated as of September 30, 1994 between
The First National Bank of Boston and Registrant.
10.48++ Lease Agreement between the Registrant and Rhode Island
Industrial Facilities Corporation, dated as of August 1,
1992.
10.49++ First Amendment to Lease Agreement between Registrant and
The Rhode Island Industrial Facilities Corporation dated as
of September 15, 1994.
10.50++ Supplementary Agreement dated as of July 1, 1994 between
Akzo Nobel Faser AG and the Registrant.
10.51**++++ Development, Marketing and License Agreement, dated as of
March 30, 1995 between Registrant and Astra AB.
10.52++++ Form of Unit Purchase Agreement to be executed by the
purchasers of the Common Stock and Warrants offered in April
1995.
10.53+++ Form of Common Stock Purchase Agreement to be executed among
the Registrant and certain purchasers of the Registrant's
Common Stock.
10.54!** Research and Commercialization Agreement dated as of
September 4, 1995 among the Company, Dr. Patrick Aebischer
and Canton of Vaud, Switzerland.
10.57!! Convertible loan agreement dated as of July 10, 1996 between
the Company and Modex Therapeutiques SA.
10.58### Lease Agreement dated as of November 21, 1997 by and between
Hub RI Properties Trust, as Landlord, and CytoTherapeutics,
Inc., as Tenant.
10.59!! Modex Therapeutiques SA stockholders voting agreement dated
as of July 10, 1996 among Modex, the Company, the Societe
Financiere Valoria SA and the other stockholders listed
therein.
10.60!! CTI individual stockholders option agreement dated as of
July 10, 1996 among the Company and the individuals listed
therein.
10.61!! CTI Valoria option agreement dated of July 10, 1996 between
the Company and the Societe Financiere Valoria SA.
10.64!!! Term Loan Agreement dated as of October 22, 1996 between The
First National Bank of Boston and the Registrant.
10.65*** Agreement and Plan of Merger dated as of August 13, 1997
among StemCells, Inc., the Registrant and CTI Acquisition
Corp.
10.67*** Consulting Agreement dated as of September 25, 1997 between
Dr. Irving Weissman and the Registrant.
10.68### Letter Agreement among each of Dr. Irving Weissman and Dr.
Fred H. Gage and the Registrant.
10.69** Amended and Restated Cross License Agreement dated as of
October 29, 1997 between Modex Therapeutiques SA and the
Registrant.
10.70### Letter Agreement dated as of September 30, 1997 between Dr.
Seth Rudnick and the Registrant.
10.71**** StemCells, Inc. 1996 Stock Option Plan.
5055
EXHIBIT NO. TITLE OR DESCRIPTION
----------- --------------------
10.72**** 1997 StemCells Research Stock Option Plan (the "1997 Plan").
10.73**** Form of Performance-Based Incentive Option Agreement issued
under the 1997 Plan.
10.74### Employment Agreement dated as of September 25, 1997 between
Dr. Richard M. Rose and the Registrant.
10.75### Employment agreement dated as of April 17, 1997, between
John S. McBride and the Registrant.
10.78### Loan Agreement dated as of May 15, 1996 between Fleet
National Bank and the Registrant, together with the related
Promissory Note executed by the Registrant, and an
amendatory agreement dated as of May 15, 1997.
10.79'10.79[*>] Rights Agreement, dated as of July 27, 1998 between Bank
Boston, N.A. as Rights Agent and the Registrant.*
10.80Section** Employment Agreement dated as of June 8, 1998 between Philip
K. Yachmetz and the Registrant.
10.81Section** Consulting Services Agreement dated as of July 27, 1998, as
amended December 19, 1998 between Dr. John J. Schwartz and
the Registrant.
10.82Section** Letter Agreement dated as of December 19, 1998 between
John J. Schwartz and the Registrant.
10.83Section** License Agreement dated as of October 27, 1998 between The
Scripps Research Institute and the Registrant.
10.84Section** License Agreement dated as of October 27, 1998 between The
Scripps Research Institute and the Registrant.
10.85Section** License Agreement dated as of November 20, 1998 between The
Scripps Research Institute and the Registrant.
10.87SectionSection** Purchase Agreement and License Agreement dated as of
December 29, 1999 between Neurotech S.A. and the Registrant.
10.88** License Agreement dated as of June 1999 between The Scripps
Research Institute and the Registrant.
10.89** License Agreement dated as of June 1999 between The Scripps
Research Institute and the Registrant.
10.90 Employment Agreement dated as of June 8, 1998, as amended
and restated as of June 8, 1999, between Philip K. Yachmetz
and the Registrant.
10.91 Letter Agreement dated as of July 1, 1999 between John J.
Schwartz and the Registrant.
10.92 Severance Agreement dated as of April 2, 1999 between John
McBride and the Registrant.
10.93 Severance Agreement dated as of August 30, 1999 between
Moses Goddard, M.D. and the Registrant.
10.9410.95 Employment Agreement dated as of November 17, 1999 between
George W. Dunbar Jr. and the Registrant.
10.9510.96 Agreement dated as of November 17, 1999 between iCEO, LLC
and the Registrant.
21 Subsidiaries of the Registrant.
23.1 Consent of Ernst & Young LLP, Independent Auditors.
27 Financial Data Schedule for fiscal year ended December 31,
1999.
99 Cautionary Factors Relevant to Forward-Looking Information.
5156
- ------------------------
++ Previously filed with the Commission as Exhibits to, and
incorporated herein by reference to, the Registrant's
Registration Statement on Form S-1, File No. 33-85494.
+++ Previously filed with the Commission as Exhibits to, and
incorporated herein by reference to, the Registrant's
Registration Statement on Form S-3, File No. 33-97272.
++++ Previously filed with the Commission as Exhibits to, and
incorporated herein by reference to, the Registrant's
Registration Statement on Form S-1, File No. 33-91228.
* Previously filed with the Commission as Exhibits to, and
incorporated herein by reference to, Registration
Statement on Form S-1, File No. 33-45739.
# Previously filed with the Commission as Exhibits to, and
incorporated herein by reference to, the Registrant's
Annual Report on Form 10-K for fiscal year ended
December 31, 1992 and filed March 30, 1993.
** Confidential treatment requested as to certain portions. The
term "confidential treatment" and the mark "**" as used
throughout the indicated Exhibits mean that material has
been omitted and separately filed with the Commission.
## Previously filed with the Commission as Exhibits to, and
incorporated herein by reference to, the Registrant's
Quarterly Report on Form 10-Q for the quarter ended
March 31, 1994 and filed on May 14, 1994.
+ Previously filed with the Commission as Exhibits to, and
incorporated herein by reference to, the Registrant's
Annual Report on Form 10-K for the fiscal year ended
December 31, 1993 and filed on March 30, 1994.
! Previously filed with the Commission as an Exhibit to and
incorporated by reference to, the Registrant's Quarterly
Report on Form 10-Q for the quarter ended March 31, 1996.
!! Previously filed with the Commission as an Exhibit to and
incorporated by reference to, the Registrant's Quarterly
Report on Form 10-Q for the quarter ended September 30,
1996.
!!! Previously filed with the Commission as an Exhibit to, and
incorporated herein by reference to, the Registrant's
Annual Report on Form 10-K for the fiscal year ended
December 31, 1996 and filed on March 31, 1997.
!!!! Previously filed with the Commission as an Exhibit to, and
incorporated herein by reference to, the Registrant's
Annual Report on Form 10-K for the fiscal year ended
December 31, 1995.
*** Previously filed with the Commission as Exhibits to, and
incorporated herein by reference to, the Registrant's
Quarterly Report on Form 10-Q for the quarter ended
September 30, 1997 and filed on November 14, 1997.
**** Previously filed with the Commission as Exhibits to, and
incorporated herein by reference to, the Registrant's
Registration Statement on Form S-8, File No. 333-37313.
### Previously filed with the Commission as an Exhibit to, and
incorporated herein by reference to, the Registrant's
annual report on Form 10-K for the fiscal year ended
December 31, 1997 and filed on March 30, 1998.
[*] Previously filed with the Commission as an Exhibit to, and
incorporated herein by reference to, the Registrant's
current report on Form 8-K filed on August 3, 1998.
Section Previously filed with the Commission as an Exhibit to, and
incorporated herein by reference to, the Registrant's
annual report on Form 10-K for the fiscal year ended
December 31, 1998 and filed on March 31, 1999.
SectionSection Previously filed with the Commission as an Exhibit to, and
incorporated herein by reference to, the Registrant's
current report on Form 8K on January 14, 2000.
(b) Current Reports on Form 8-K.
None
5257
SIGNATURES
Pursuant to the requirements of Section 13 or 15(d) of the Securities
Exchange Act of 1934, the Registrant has duly caused this report to be signed on
behalf by the undersigned, thereunto duly authorized.
CYTOTHERAPEUTICS,STEMCELLS, INC.
BY: /S/ GEORGE W. DUNBAR, JR.
-----------------------------------------
George W. Dunbar, Jr.
ACTING PRESIDENT AND CHIEF EXECUTIVE
OFFICER
Dated: April 14,December 5, 2000
Pursuant to the requirements of the Securities Exchange Act of 1934, this
report has been signed below by the following persons on behalf of the
Registrant and in the capacities and on the dates indicated.
SIGNATURE CAPACITY DATE
--------- -------- ----
Acting President And Chief
/s/ GEORGE W. DUNBAR, JR. Executive Officer
And Acting
------------------------------------------- Chief Financial Officer April 14,(principal executive December 5, 2000
George W. Dunbar, Jr. officer)
Controller and Acting Chief
/s/ GEORGE KOSHY Financial Officer
------------------------------------------- (principal executivefinancial December 5, 2000
George Koshy officer and principal
accounting officer)
/s/ MARK J. LEVIN Director
------------------------------------------- April 14,December 5, 2000
Mark J. Levin
/s/ DONALD KENNEDY, PH.D. Director
------------------------------------------- April 14,December 5, 2000
Donald Kennedy, Ph.D.
/s/ JOHN J. SCHWARTZ, PH.D. Director, Chairman of the
------------------------------------------- Board ------------------------------------------- April 14,December 5, 2000
John J. Schwartz, Ph.D.
/s/ IRVING L. WEISSMAN, M.D. Director
------------------------------------------- April 14,December 5, 2000
Irving L. Weissman, M.D.
53
EXHIBIT 99
CAUTIONARY FACTORS RELEVANT TO FORWARD-LOOKING INFORMATION
CYTOTHERAPEUTICS, INC. (THE "COMPANY" OR "WE" OR "US") WISHES TO CAUTION
READERS THAT THE FOLLOWING IMPORTANT FACTORS, AMONG OTHERS, IN SOME CASES HAVE
AFFECTED AND IN THE FUTURE COULD AFFECT THE COMPANY'S RESULTS AND COULD CAUSE
ACTUAL RESULTS AND THE NEEDS AND FINANCIAL CONDITION OF THE COMPANY TO VARY
MATERIALLY FROM FORWARD-LOOKING STATEMENTS MADE BY THE COMPANY ON THE BASIS OF
MANAGEMENT'S CURRENT EXPECTATIONS. THE BUSINESS IN WHICH THE COMPANY IS ENGAGED
IS DEPENDENT ON UNPROVEN TECHNOLOGY, RAPIDLY CHANGING, EXTREMELY COMPETITIVE AND
INVOLVES A HIGH DEGREE OF RISK, AND ACCURACY WITH RESPECT TO FORWARD-LOOKING
STATEMENTS IS DIFFICULT.
Investment in CytoTherapeutics involves a high degree of risk. Some of the
following risks relate principally to our business and the industry in which we
operate. Other risks relate principally to the securities market and ownership
of our stock. The risks described below are not the only ones facing us.
Additional risks, not now known to us or that we currently believe to be
immaterial, may also adversely affect our business. Our business, financial
condition or results of operation could be materially or adversely affected by
any of these risks. The trading price of our stock could decline due to any of
these risks, and investors may lose all or part of their investment.
OUR TECHNOLOGY IS AT AN EARLY STAGE OF DEVELOPMENT.
Our stem cells technology is at the early pre-clinical stage and has not yet
led to the development of any proposed product. There can be no assurance that
our stem cell technology will lead to the development of any proposed product or
that any product that may be developed in the future in our stem cell programs
will (i) survive and persist in the desired location, (ii) provide the intended
therapeutic benefits, (iii) properly differentiate and integrate into existing
tissue in the desired manner, or (iv) not cause side effects. Results obtained
in early pre-clinical research may not be indicative of the results that will be
obtained in later stages of pre-clinical or clinical research.
Issues we do not now face because the technology is at an early stage may
become relevant in the future. For example, we neither have nor need marketing,
sales or distribution capabilities. When and if we develop a product or
products, we will need to develop such capabilities or acquire them through
licensing or other relationships with partners. There can be no assurance that
we will be able either to develop adequate capabilities in-house or to enter
into technically and financially acceptable relationships to acquire them. Since
stem cells represent a novel form of therapy, there can be no assurance that any
products we may develop will be accepted in the marketplace. Moreover, the use
of such novel technology could expose us to product liability claims; we cannot
be certain that we will be able to obtain adequate liability insurance on
commercially reasonable terms or otherwise protect ourselves from such claims.
WE HAVE LIMITED LIQUIDITY AND CAPITAL RESOURCES AND MUST OBTAIN SIGNIFICANT
CAPITAL RESOURCES IN ORDER TO SUSTAIN OUR RESEARCH AND DEVELOPMENT EFFORTS.
We have limited liquidity and capital resources and must obtain substantial
additional capital in order to sustain research and development efforts.
Substantial additional funds will be required to support our research and
development programs, for acquisition of technology and intellectual property
rights, and, to the extent we decide to undertake these activities ourselves,
for pre-clinical and clinical testing of our anticipated products, pursuit of
regulatory approvals, establishment of production capabilities and general
administrative expenses.
We intend to pursue our needed capital resources through equity and debt
financings, corporate alliances, grants and collaborative research arrangements.
Our ability to complete any such arrangements successfully will depend upon
market conditions and, more specifically, on our progress in our research and
development efforts. There can be no assurance that we will obtain the necessary
capital resources from any such sources. Lack of necessary funds may require us
to delay, reduce or eliminate some or all of
54
our research and development programs or to license our technology or any
potential products resulting therefrom to third parties. There can be no
assurance that funding will be available to us when needed, if at all, or on
terms acceptable to us.
WE MAY NEED TO OBTAIN A PARTNER OR PARTNERS TO SUPPORT OUR STEM CELL
DEVELOPMENT EFFORTS.
Equity and other funds alone may not be sufficient to fund the cost of
developing our stem cell technologies and we may be dependent on our ability to
reach appropriate partnering arrangements providing support for our stem cell
discovery and development efforts. While we have engaged, and expect to continue
to engage, in discussions regarding such arrangements, we have not reached any
agreement regarding any such arrangement and there can be no assurance that we
will be able to obtain any such agreement on terms acceptable to us, if at all.
WE HAVE A HISTORY OF OPERATING LOSSES AND THERE CAN BE NO ASSURANCE THAT WE
WILL DEVELOP PRODUCTS, OBTAIN PRODUCT REVENUES OR BECOME PROFITABLE.
Substantially all of our revenues to date have been derived from, and, for
the foreseeable future, substantially all of our revenues are expected to be
derived from, cooperative agreements, research grants and income earned on
invested funds. We have incurred substantial operating losses in the past; as
the financial statements in this Form 10K indicate, we incurred a net loss of
approximately $4.568 million in the fourth quarter of 1999, resulting in our
holding approximately $4.76 million in cash and cash equivalents at the end of
1999. The Company will continue to incur substantial operating losses in the
future in order to conduct our research and development activities and, if those
are successful, to fund clinical trials and other expenses. There can be no
assurance that we will develop any product candidates, achieve revenues from any
product sales or become profitable.
WE HAVE SUBSTANTIAL OBLIGATIONS AND POTENTIAL OBLIGATIONS RESULTING FROM THE
CONDUCT OF OUR FORMER ENCAPSULATED CELL THERAPY BUSINESS.
We continue to have substantial obligations in regard to our former
encapsulated cell therapy facilities and administrative offices in Rhode Island,
including lease payments and operating costs of approximately $950,000 per year
associated with our former Science and Administration Facility ("SAF") in
Lincoln, Rhode Island, and debt service payments and operating costs of
approximately $1,000,000 per year with respect to our former encapsulated cell
therapy pilot manufacturing facility, also located in Lincoln, Rhode Island. We
are currently seeking to sublease the SAF and to sell the pilot manufacturing
facility, but there can be no assurance that we will succeed in these efforts.
Failure in these efforts within a reasonable time period could have a material
adverse effect on our liquidity and capital resources and adversely affect our
ability to fund further development of our stem cell technology.
THERE CAN BE NO ASSURANCE THAT WE WILL REALIZE ANY FURTHER REVENUE FROM THE
SALE OF OUR ENCAPSULATED CELL TECHNOLOGY.
In December 1999, we sold our encapsulated cell therapy technology to
Neurotech S.A. While the sale provides for the possibility of our receiving
royalty and other payments from Neurotech, there can be no assurance that any
such payments will be received and we do not anticipate receiving any material
payments from Neurotech in the near future, if at all.
FOUNDERS OF STEMCELLS CALIFORNIA, INC. HAVE THE RIGHT TO CONTROL OUR STEM
CELL RESEARCH AND TO REACQUIRE OUR STEM CELL TECHNOLOGY UNDER CERTAIN
CIRCUMSTANCES.
The agreement by which CytoTherapeutics acquired StemCells California in
September 1997, provides for our stem cell research to be conducted pursuant to
the provisions of an agreement between CytoTherapeutics and Drs. Irving Weissman
and Fred Gage, founders of StemCells California, pursuant to a research plan.
For so long as the goals of the research plan are accomplished, the stem cell
research will continue to be conducted under an extension of such research plan
approved by a Research Committee consisting of two persons chosen by Drs. Gage
and Weissman, two chosen by CytoTherapeutics, and a fifth
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member appointed by Drs. Gage and Weissman, subject to the reasonable approval
of CytoTherapeutics. Increases in stem cell research funding of not more than
25% a year approved by the Committee must be funded by CytoTherapeutics as long
as the goals of the research plan are being met, provided, however, that
CytoTherapeutics will retain the option of ceasing or reducing neural stem cell
research even if all research plan goals are being met by accelerating the
vesting of all still-achievable performance-based options granted to Drs.
Weissman and Gage in connection with the acquisition of StemCells. If we cease
or reduce non-neural stem cell research although all research plan goals are
being met, however, Drs. Weissman and Gage would have the right to continue
development of the non-neural stem cell research by licensing the technology
related to such research to the founders in exchange for a payment to
CytoTherapeutics equal to all funding for such research, plus royalty payments,
which might not reflect fair market value for such technology at such time.
PATENT PROTECTION FOR OUR TECHNOLOGY IS IMPORTANT BUT UNCERTAIN.
Patent protection for products such as those we propose to develop is highly
uncertain and involves complex factual and legal questions. There can be no
assurance that any of our current or future patent rights will not be
challenged, invalidated or circumvented, or that the rights granted under any
such patent will provide competitive advantages to us. Because the first person
or entity to discover and obtain a valid patent to a particular stem or
progenitor cell may effectively block all others, it will be important to our
development efforts for us or our collaborators to be the first to discover any
stem cell that we are seeking; failure to do so could have a materially adverse
effect on the Company.
Proprietary trade secrets and unpatented know-how are also important to our
research and development activities. We cannot be certain that others will not
develop the same or similar technologies on their own, or that we will be able
to protect our trade secrets and unpatented know-how and to keep them secret.
WE MAY NEED TO OBTAIN LICENSES TO THIRD PARTY PATENTS.
A number of pharmaceutical companies, biotechnology and other companies,
universities and research institutions have filed patent applications or have
been issued patents related to cell therapy and other technologies potentially
relevant to or required by our potential products. We cannot predict which, if
any, of any such applications will issue as patents or the claims that might be
allowed. We are aware that a number of entities have filed applications relating
to stem and/or progenitor cells. We cannot predict the effect of existing patent
applications and patents on our technology or any future products we may
develop. We may be required to seek licenses from others in order to
commercialize our technology. There can be no assurance that we will be able to
obtain such licenses on acceptable terms, if at all, or that the patents
underlying any such licenses will be valid and enforceable.
DEVELOPMENT OF OUR TECHNOLOGY WILL BE SUBJECT TO EXTENSIVE GOVERNMENT
REGULATION.
Our research and development efforts, as well as any future clinical trials,
and the manufacturing and marketing of any products we may develop, will be
subject to extensive regulation by governmental authorities in the United States
and other countries. The process of obtaining FDA and other required regulatory
approvals is lengthy, expensive and uncertain. There can be no assurance that we
or our collaborators will be able to obtain the necessary approvals to commence
or continue clinical testing or to manufacture or market our potential products
in reasonable anticipated time frames, if at all. In addition, the United States
Congress and other legislative bodies may enact regulatory reforms or
restrictions on the development of new therapies that could adversely affect the
regulatory environment in which we operate or the development of any products we
may develop.
DEVELOPMENT OF OUR STEM CELL TECHNOLOGY MAY BE MATERIALLY ADVERSELY AFFECTED
BY REGULATORY CONCERNS REGARDING CELL THERAPY.
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Regulatory concerns regarding the risk of cell transplantation could lead to
restrictions on the testing or use of cells as human therapeutics, which could
materially adversely affect our stem cell development programs and the Company
itself and could prevent us from developing, producing, licensing or selling
products or make the cost of developing or producing products prohibitively
high.
ACQUISITION OF NEEDED CELLS AND OTHER MATERIALS MAY BE DIFFICULT.
There can be no assurance that the Company will successfully identify or
develop reliable and ethical sources of the cells required for its potential
products and obtain such cells in quantity and quality sufficient to satisfy the
commercial requirements of its potential products.
WE ARE SIGNIFICANTLY DEPENDENT ON A LIMITED NUMBER OF KEY PERSONNEL.
We are highly dependent on the principal members of our management and
scientific staff and certain of our outside consultants. Loss of the services of
any of these individuals could have a material adverse effect on our operations.
In addition, our operations are dependent upon our ability to attract and retain
additional qualified scientific and management personnel. There can be no
assurance the Company will be able to attract and retain such personnel on
acceptable terms given the competition among pharmaceutical, biotechnology and
health care companies, universities and research institutions for experienced
personnel.
WE MAY BE SIGNIFICANTLY DEPENDENT ON THIRD PARTIES.
In order to successfully develop and commercialize our technology, we may
need to enter into a wide variety of arrangements with corporate sponsors,
pharmaceutical companies, universities, research groups and others. There is no
assurance that will be able to establish and maintain such arrangements on terms
acceptable to us, or to successfully perform our obligations under such
arrangements. If any of our collaborators terminates its relationship with us or
fails to perform its obligations in a timely manner, the development or
commercialization of our technology and any product candidates we may develop
may be adversely affected.
WE MAY NOT BE ABLE TO KEEP PACE WITH TECHNOLOGICAL CHANGE OR WITH THE
ADVANCES OF OUR COMPETITORS.
Competitors of the Company are numerous and include major pharmaceutical and
chemical companies, biotechnology companies, universities and other research
institutions. In addition, we have competitors with substantially greater
capital resources, experience in obtaining regulatory approvals and, in the case
of commercial entities, experience in manufacturing and marketing pharmaceutical
products, than we do. There can be no assurance that our competitors will not
succeed in developing technologies and products that are more effective than
those being developed by CytoTherapeutics or that would render our technology
and products obsolete or non-competitive.
HEALTHCARE INSURERS AND OTHER ORGANIZATIONS MAY NOT PAY FOR OUR PRODUCTS OR
MAY IMPOSE LIMITS ON REIMBURSEMENTS.
In both domestic and foreign markets, sales of potential products is likely
to depend in part upon the availability and amounts of reimbursement from third
party health care payor organizations, including government agencies, private
health care insurers and other health care payors such as health maintenance
organizations and self-insured employee plans. There is considerable pressure to
reduce the cost of therapeutic products. There can be no assurance that
reimbursement will be provided by such payors at all or without substantial
delay, or, if such reimbursement is provided, that the approved reimbursement
amounts will provide sufficient funds to enable us to sell products we may
develop on a profitable basis. Changes in reimbursement policy could also
adversely affect the willingness of pharmaceutical companies to collaborate with
the Company on the development of our stem cell technology.
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OUR QUARTERLY OPERATING RESULTS MAY FLUCTUATE.
Our operating results have varied, and may in the future continue to vary,
significantly from quarter to quarter due to a variety of factors. These factors
include the receipt of one-time license or milestone payments under
collaborative agreements, costs associated with termination of our encapsulated
cell therapy business, variation in the level of expenses related to our
research and development efforts, receipt of grants or other support for our
research and development efforts, and other factors. Quarterly comparisons of
our financial results are not necessarily meaningful and should not be relied
upon as an indication of future performance. These fluctuations may cause the
price of our stock to fluctuate, perhaps substantially.
OUR STOCK PRICE MAY BE VOLATILE.
The market price for our Common Stock has been volatile and could decline
below the offering price for the shares. We believe that the following factors,
among other things, have caused the market price for our Common Stock to
fluctuate substantially, and that they will continue to do so in the future:
- The results of scientific developments by us or our competitors;
- The formation or termination of corporate alliances;
- Determinations regarding our patent rights and the patent rights of
others; and
- Variations in our quarterly operating results.
The stock market has recently experienced extreme price and volume
fluctuations. These fluctuations have especially affected the market price of
the stock of many high technology and health care-related companies. Such
fluctuations have often been unrelated to the operating performance of these
companies. Nonetheless, these broad market fluctuations may negatively affect
the market price of our Common Stock.
EVENTS WITH RESPECT TO OUR SHARE CAPITAL COULD CAUSE THE PRICE OF OUR COMMON
STOCK TO DECLINE.
Sales of substantial amounts of our Common Stock on the open market, or the
availability of such shares for sale, could adversely affect the price of our
Common Stock. In particular, as of March 20, 2000, stock options to purchase
approximately 965,160 shares of Common Stock were outstanding, at an average
exercise price of approximately $2.281 per share (subject to adjustment in
certain circumstances); of this total, options covering approximately 395,811
shares are currently exercisable at an average exercise price of approximately
$3.456 per share.
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