BIOPHARMA

Stem Cell Companies: Burning Cash on Research with Hopes of New Drugs and Therapies

Michael A. Wijaranakula
Fri Sep 9, 2005

Geron is also developing cell-based therapeutics based on differentiated cells derived from hESCs including neural cells to repair spinal cord injuries, dopaminergic neurons that are lost because of Parkinson’s disease, cardiomyocytes for heart disease, pancreatic islet ß cells for type 1 diabetes, chondrocytes for osteoarthritis, osteoblasts for osteoporosis, and hematopoietic stem cells for blood diseases and to prevent immune rejection of the other cell types. Geron plans to begin clinical trials on acute spinal cord injury treatment in early 2006, according to chief executive officer Tom Okarma. 

Recently, research scientists at the University of Washington, in collaboration with Geron, successfully transplanted human embryonic stem cells into the rodent heart thereby creating new blood vessels around the hearts. "The successful engraftment of the hESC-derived cardiomyocytes provides evidence for the feasibility of using these cells in myocardial repair," stated Jane S. Lebkowski, Ph.D., Geron's senior vice president of regenerative medicine.

Last quarter, Q2 2005, Geron reported revenues of $4.7 million with a net loss of $3.1 million or $(0.06) per share, compared to revenues of $366,000 and losses of $8.9 million or $(0.20) per share in the second quarter of 2004. A marked increase in quarterly revenue was due to an upfront payment of $4 million in conjunction with the transfer of intellectual property rights related to nuclear transfer for use in animal cloning to the company's new joint venture, stART Licensing, Inc. 

The Research and Development (R&D) expenses of $6.8 million for Q2 2005 decreased compared to operating expenses of $9.5 million in 2004 due to reductions in costs to produce supplies of GRN163L. Geron expects R&D expenses to increase in the next year as the company incurs expenses related to clinical trials of telomerase inhibitor, GRN163L, continued clinical trials of their telomerase cancer vaccine and continued development of their hESC programs.

Geron's share price soared 26.6 percent in July of this year after the company announced a collaborative agreement with Merck (NYSE:MRK) on development of cancer vaccines. We are expecting additional positive announcements from Geron in the coming quarters regarding their therapeutic vaccine targeting telomerase in patients with metastatic prostate cancer and cell-based therapies which could drive the share price even higher in the next 12 months. 


StemCells Inc. (NASDAQ: STEM), a Palo Alto, CA-based biotechnology company, is focused on stem cell-based therapies to treat diseases and disorders of the central nervous system (CNS), as well as diseases of the liver and pancreas. StemCells Inc., has isolated human central nervous system stem cells (hCNS-SC) directly from normal brain tissue and has shown that these adult stem cells can migrate to different regions of the brain to become specialized cells as a potential treatment for Batten disease, spinal cord injury, demyelinating diseases, stroke and Alzheimer's disease.

StemCells Inc.'s patent portfolio in the stem cell therapy area includes 43 U.S. patents issued. Their proprietary human neural stem cells, HuCNS-SC, had its IND application filed in December 2004 for the treatment of Batten disease, a genetic disease causing neurological cell loss. The Investigational New Drug (IND) application is currently on clinical hold to respond to the Food & Drug Administration’s (FDA) questions and concerns. In late August 2005, the State of California granted StemCells, Inc. a manufacturing license for its cell processing facility for HuCNS-SC. “Acquiring this license is another milestone in our progress toward beginning our first human clinical trial,” said Martin McGlynn, President and CEO of StemCells.

StemCells posted widening losses of $3.9 million for Q2 2005 versus a loss of $3.3 in the same period last year. Revenues grew from $6,000 for the period last year to $36,000 for the quarter in 2005, primarily from grants and licensing. R&D spending for the quarter was $2.1 million, compared with $1.9 million for the same period in 2004, primarily due to an increase in the number of full-time employees, now 30 compared to 24 in 2004, working in R&D and lab support.

Aastrom Biosciences, (NASDAQ:ASTM) based in Ann Arbor, MI with a current market cap of $280 million, develops products for the repair or regeneration of human tissues utilizing its proprietary adult stem cell technology to focus on Tissue Repair Cells (TRCs), a mix of bone marrow-derived adult stem and progenitor cells. Aastrom is enrolling participants for Phase I/II level clinical trials in developing TRCs for the treatment of severe bone fractures, ischemic vascular disease, jaw reconstruction, and spine fusion. Aastrom's total annual revenues for 2004 were $1.3 million.

Although StemCells' and Aastrom Biosciences' shares are among best performers of the stem cell-related group in the past 12 months with a return of over 200 percent, compared to the 30 percent return of the AMEX Biotechnology Index (AMEX:$BTK.X), investors should be aware that the average daily trading volume of StemCells, 3.1 million shares, and Aastrom Bioscience, 3.4 million shares, are extremely high for companies with a market cap of just under $350 million. Heavy trading days for stem cell companies such as STEM and ASTM are activities most likely driven by day-trading and short-selling.

ViaCell Inc., (NASDAQ: VIAC) based in Cambridge, MA, with Q2-2005 revenues of $11 million, is a biotech company currently working with umbilical cord blood and stem cell therapy. Their Viacord unit, which provides almost all of their revenue, is a leading private cord blood bank that collects and cryopreserves umbilical cord blood for customers. Thus far, Viacord stores over 63,000 cord blood units and reports that cord blood retains viability and function for 15 years or more when stored under cryopreservation.

ViaCell hopes to enable applications of hematopoietic (blood) stem cells from umbilical cord blood with their lead cellular therapy product candidate, CB001, for use in bone marrow transplants. CB001 is currently in Phase I clinical trials with reports indicating Phase II studies are to begin in mid-2006.

ViaCell recently announced an expansion of its collaboration and license agreement with Amgen (NASDAQ: AMGN) and new license from Johns Hopkins for the use of growth factors for use in developing and manufacturing cellular therapy products. "Amgen's r-metHuG-CSF is an important growth factor with demonstrated utility in cellular therapies.

By applying r-metHuG-CSF to the Johns Hopkins co-culturing technology, we believe we will be able to extend our current cell expansion platform for the manufacture of next-generation hematopoietic stem cell products," said Marc Beer, president and CEO of ViaCell. "We believe that this is further indication of ViaCell's ability to identify the technologies that could ultimately help realize the therapeutic value of cord blood stem cells."

Financial results for ViaCell for Q2 2005 report that total revenues were $11.4 million compared with $9.7 million for the same period in 2004 with Q2 2005 net losses widening to $3.1 million, compared to a net loss of $2.3 million in the same period in 2004.R&D expenses are $3.0 million for the latest quarter, compared with $3.9 million for the second quarter in 2004, attributed to the closure of their German laboratory but offset in the increase in clinical development expenses related to the Phase I trial of CB001.

VIAC share prices have been virtually flat since its Initial Public Offering (IPO) in late January this year. Although VIAC shares has dropped as much as 35% from its IPO pricing of $7.00/ share, Wall Street analysts are expecting VIAC's revenue to grow as much as 22% next year and set a price target as high as $15.00. 

Less Risky Investment. Companies providing specialized biotech R&D products, equipment and services, such as Invitrogen Corporation (NASDAQ: IVGN), may be less risky than the stem cell companies themselves with customers including corporate, academic and government research facilities. Invitrogen Corporation, with annual revenues of over $1 billion, is a Carlsbad, CA-based global provider of products and services for the life science research market with its BioDiscovery and BioProduction segments. The segments cover functional genomics, cell biology, and drug discovery product lines, software to analyze and interpret genomic, proteomic, and other biomolecular data as well as various cell culture products and biological testing services.

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