Investor FAQs
Q: What does StemCells do?
A: StemCells, Inc. is engaged in the discovery and development of adult stem cells to treat diseases of, or injury to, the central nervous system (CNS), liver and pancreas. The Company is among the most advanced in pursuing stem cell therapies, and uses a proven search methodology to isolate rare stem and progenitor cells from human tissues. Stem cells are cells that can produce all of the types of mature cells of an organ and can also self-renew; progenitor cells are cells that come from stem cells, can still produce one or more types of mature cells in an organ, but have limited or no self renewal capacity. To date, StemCells has found the human neural stem cell, a candidate human liver stem cell and a candidate pancreas stem cell. The Company has either issued patents or pending patent applications on all three cell types.
A number of diseases, including neurodegenerative disorders, paralysis and type I diabetes, are associated with the death of specific cell populations that do not regenerate sufficiently to replace lost cells. Stem cells derived from a particular organ, when transplanted into that organ, may provide an ongoing supply of the specialized functional cells of that organ. In October 2005, the Company received clearance from the US Food and Drug Administration to initiate a Phase I clinical trial to evaluate the safety and preliminary efficacy of the Company's human neural stem cells (HuCNS-SCT™) as a treatment of neuronal ceroid lipofuscinosis (NCL, also referred to as Batten disease), a rare, fatal neurodegenerative lysosomal storage disorder. In March 2006, the Company received approval from the Institutional Review Board (IRB) at the Oregon Health & Science University to begin the Phase I clinical trial at OHSU Doernbecher Children's Hospital in Portland, Oregon. Both clearance by the FDA and an IRB were required in order to initiate the clinical trial.For more information on the Company, please see our corporate fact sheet.
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Q: What is Batten Disease?
A: Batten disease is named after the British pediatrician who first described the juvenile form of neuronal ceroid lipofuscinosis (NCL) in 1903. The name is now commonly used to encompass three forms of NCL-infantile, late infantile and juvenile onset. All forms have the same basic cause-lack of a lysosomal enzyme-and have a similar progression and outcome. The different forms of NCL have traditionally been classified by age of onset. Now with genetic testing, the disease is more precisely classifiable in terms of mutations in the specific gene and enzyme causing the disease. Children with Batten disease suffer seizures, progressive loss of motor skills, sight and mental capacity, eventually becoming blind, bedridden and unable to communicate. Today, Batten disease is always fatal.
Infantile and late infantile NCL are brought on, respectively, by inherited mutations in the CLN1 gene, which codes for palmitoyl-protein thioesterase 1 (PPT1) or in the CLN2 gene, which codes for tripeptidyl peptidase I (TPP-I). The consequence of these mutations is either a defective or a missing enzyme that leads to accumulation of lipofuscin-like fluorescent inclusions in various cell types. Presumably, these non-degraded lysosomal substrates accumulate to the point where they interfere with normal cellular and tissue function and ultimately lead to the pathological manifestations of the disease. One way to treat the disease may be to provide the brain with a replacement source of functional enzyme that can be taken up by the enzyme-deficient cells.
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Q: What is the goal of StemCells' Phase I trial in Batten disease?
A: The Phase I trial is designed to evaluate the safety and preliminary efficacy of HuCNS-SC for the treatment of infantile and late-infantile Batten disease. In addition to measuring the safety of HuCNS-SC, the trial will provide initial data on HuCNS-SC's ability to affect the progression of the disease. Children enrolled in the study will be evaluated with standardized measures of development, cognition, behavior and language for one year following HuCNS-SC transplantation. Patients will also be asked to permit monitoring to be continued for at least a five year period after transplantation
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Q: When will the Company's Batten trial begin?
A: In order to initiate a clinical trial in the US, both clearance by the FDA and an IRB are required. The Company received clearance from the FDA to initiate the Phase I clinical trial in Batten disease in October 2005, and in March 2006 received approval from the IRB at the Oregon Health & Science University to proceed at OHSU Doernbecher Children's Hospital in Portland, Oregon.
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Q: How does the Company currently fund its research programs?
A: Our R&D programs, all of which concentrate on diseases of, or injury to, the central nervous system, the liver and the pancreas, have been funded primarily by StemCells’ shareholders, rather than by the public sector. As of December 31, 2005, the Company had $34.5 million in cash and cash equivalents.
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Q: When was StemCells incorporated, and in which state?
A: The Company now known as StemCells was incorporated in Delaware in August 1988, under the name Cellular Transplants, Inc. In February, 1992 the Company was renamed CytoTherapeutics and in April of that year the Company had its initial public offering. In May 2000, the Company changed its name to StemCells, Inc., reflecting its decision to focus solely on stem cell therapies .
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Q: How many people does StemCells employ?
A: As of December 31, 2006, StemCells employed 46 people.
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Q:
How does California’s Proposition 71 affect StemCells?
A: In November 2004, California State Proposition 71 (Prop. 71), the California Stem Cell Research and Cures Initiative, was adopted by the electorate. It is intended to encourage stem cell research in the State of California, and to finance such research with State funds of approximately $295 million annually for 10 years beginning with 2005. It is our understanding that the California Institute for Regenerative Medicine created under the Initiative will provide grants, primarily but not exclusively to academic institutions, to advance both embryonic stem cell research and adult stem cell research; the latter is the current and exclusive focus at StemCells. We are eligible to receive Prop. 71 generated funds and we do intend to apply for such funding; no funding is now available, however, pending resolution of legal issue challenges to Prop 71.
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Q:
Is StemCells eligible to receive funds under Prop. 71?
A: As a company operating in California since 1995 and as a leader in tissue-derived stem cells, StemCells is eligible to receive Prop. 71 generated funds and we do intend to apply for additional funding; no funding is available, however, pending resolution of legal issue challenges to Prop 71. We also remain eligible for federal government support from the National Institutes of Health (NIH) due to our focus on adult stem cells. NIH grants to the Company or to academic collaborators have assisted and continue to assist research in the use of our cells for various diseases and conditions such as Alzheimer's disease and spinal cord injuries. The Company considers such grants to be important confirmation of the quality of its science and intellectual property, but does not rely on them as a significant source of funding.
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Q: What is StemCells' fiscal year end?
A: December 31.
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Q: On which exchange does StemCells trade and what is the symbol?
A: StemCells trades on the NASDAQ National Market. The ticker is STEM.
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Q: When did StemCells become public?
A: The Company went public in 1992.
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Q: How many shares are outstanding?
A: As of March 7, 2006, there were 65,402,682 shares of Common Stock issued and outstanding.
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Q: Does StemCells issue dividends on its common stock?
A: No.
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Q: Does StemCells have a direct stock purchase plan?
A: No.
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Q: Who are StemCells' independent auditors?
A: Grant Thornton LLP is the Company's independent auditor.
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Q: Who is StemCells' stock transfer agent?
A: Computer Share Investor Services
P.O. Box 43023
Providence, RI 02940-3023
Phone: 781.575.2879
Fax: 617.360.6900
www.computershare.com
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Q: Who is StemCells' legal counsel?
A: Ken Stratton is StemCells' General Counsel. The Company's outside corporate counsel is Ropes and Gray of Boston and its patent counsel is Mintz, Levin, Cohn, Ferris, Glovsky and Popeo, also of Boston.
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Q: How do I contact StemCells' Investor Relations?
A: You can contact Investor Relations by calling 650.475.3100 extension 105 or via e-mail at ir-pr@stemcellsinc.com. Investor information can also be downloaded from our website at www.stemcellsinc.com.
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Thank you for your interest in StemCells, Inc.
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