Stem Cell Biotechs Report 2011 Year In Review: Part I

by: Chemistfrog

The biotech sector is comprised of a host of small pharmaceuticals striving to make their mark in the lives of the patients they hope to save, heal, alleviate symptoms for or help in one form or another. With the targeted patient set successfully treated, each company and its investors hope to reap huge rewards on their investments of time and money. The stem cell biotechs is a budding group within the field that hopes to address unmet needs via its many mechanisms of stem cell production and application methods. Early hope in the technology indicates progress in treating a multitude of injuries and diseases including, but not limited to, ALS (Lou Gehrig's disease), Parkinson's disease, spinal cord injury, burns, heart disease, diabetes, arthritis or virtually any condition in which regenerated tissue can heal, slow disease progression or rebuild organs. The sector has left the fledgling stage and is now maturing with Phase III clinicals now underway.

In late February and March, publicly-traded stem cell biotechs presented their financials and accomplishments for 2011 along with their goals for 2012. Investors interested in diversifying their portfolios and gaining entry-level positions into one of the most exciting sectors in the biotech field may view the recent updates and filings below to assist them in ascertaining what types of investments they might consider in this budding sector. Market capitalizations (MCAP) for each company at market close on April 27th are noted for comparative purposes. Although the sector is still developing, a notable few companies maturing with pipeline development and/or manufacturing capabilities (and revenue generation) are beginning to stand out.

Of the list below, NeoStem (NBS) and Aastrom (ASTM) are true standouts for various reasons. Both companies have recently completed financing which should allow each to progress well into their current trials. Each also possesses more than ample manufacturing capabilities with NeoStem gaining an advantage via its Progenitor division serving multiple pharmaceutical customers and its Suzhou Erye Pharmaceutical also generating revenue with the latter currently being marketed for sale. Suzhou Erye's tentative sale along with NeoStem's recent financing will give the company a firm financial footing to stand on, especially impressive for a budding biotech with a $41 million market cap. Aastrom currently leads with regard to pipeline as a Phase III trial of its Ixmyelocel-T cell therapy, termed the REVIVE trial, for critical limb ischemia (CLI) initiated in February 2012. The trial has the benefit of a Fast Track designation by the FDA along with a Special Protocol Assessment (SPA) from them as well for the trial's design. NeoStem's third division (one of two it acquired in 2011) has the company's lead product in the form of Amorcyte Inc's AMR-001 with a Phase II trial initiating in January 2012 for the treatment of acute myocardial infarction (heart attack), a potentially huge market group. AMR-001 is autologous (made the patient's own cells, in this case from their bone marrow) greatly reducing the probability of rejection by the patient's immunity system and adding to the treatment's safety profile. Showing its own manufacturing strength and expertise, NeoStem's Progenitor division will be providing manufacturing, supply and logistics for the trial.

International Stem Cell Corp. (OTCQB:ISCO), with an MCAP of $34.8 million, released its corporate update on March 20th. For the year ending December 31st, the company reported $4.5 million in revenue, an increase of 189% over 2010. The increases in revenues in both periods were reported as driven by strong sales at ISCO's wholly-owned subsidiary Lifeline Skin Care (LSC). Steady growth in sales from ISCO's other wholly-owned subsidiary, Lifeline Cell Technology (LCT), also contributed to the increase in revenue.

Two key highlights in its stem cell research program were noted:

  1. "A number of donors willing to provide oocytes for research purposed were enrolled in ISCO's program to establish a bank of clinical grade hpSC capable of being immune-matched to millions of patients.
  2. The Research and Development team successfully completed the first series of preclinical studies that supports the therapeutic use of hepatocytes (liver cells) and neuronal cells derived from human parthenogenetic stem cells (hpSC). These in vivo experiments demonstrated that the derived cells are able to survive in targeted location in mice without causing tumors."

NeoStem, Inc., with an MCAP of $41.2 million, gave its 2011 update on March 20th, 2012. The company reported consolidated revenue of $73.7 million for 2011 relative to $69.8 million for 2010. The company's consolidated net loss for 2011 was $56.6 million including $10.3 million of non-cash equity-based compensation expense, $19.4 million of goodwill impairment charges and $9.0 million of depreciation and amortization. NeoStem's consolidated cash loss for 2011 was $15.5 million (see reconciliation below). Net loss attributable to NeoStem common shareholder interests for 2011 was $47.8 million, or $0.54 per share. At year end, the company reported cash and cash equivalents of $12.7 million with an additional $2.5 million in cash held in escrow. The company's financials were additionally shored up with a $6.8 million ($6 million plus an over-allotment) offering of 15 million shares priced at $0.40 per unit with each unit consisting of a share of common stock and a warrant to purchase an additional share with an exercise price of $0.51.

The following pertinent 2011 stem cell research highlights were significant and noted:

  1. NeoStem believes that the opportunities that exist today in cell therapy are robust and growing despite a persistently difficult financial environment, making this an opportunistic time to pursue the monetization of the Company's 51% ownership of Suzhou Erye Pharmaceutical Co., Ltd. and bolster its cell therapy business. In June 2011, the Company engaged a financial advisor to lead the effort to pursue the possible divesture of the Company's interest in Erye. Marketing efforts are underway and have generated interest from both financial and strategic buyers.
  2. NeoStem is also developing pre-clinical assets, including its VSEL™ Technology platform for regenerative medicine, which NeoStem believes is an endogenous pluripotent non-embryonic cell that has the potential to change the paradigm of cell therapy as we know it today. These activities have received awards in excess of $2.5 million which funds support the work of prestigious researchers who are pioneering this science with NeoStem.
  3. Behind the development of these therapeutic assets is the NeoStem cell therapy contract manufacturing business (PCT) which itself continues to grow. New clients have engaged PCT to assist them in the development of their products, including a global, diversified healthcare company who recently selected PCT to provide stem cell processing in our two GMP manufacturing facilities in the United States (California and New Jersey). PCT's prominence in the marketplace continues to grow and that is reflected by both client satisfaction and the revenues the company generates. (Acquired January 20th, 2011).
  4. NeoStem now has a pipeline of assets that includes October 2011 acquired Amorcyte (Phase II trial for preservation of heart function after a heart attack), Athelos (physician sponsored Phase I trials for a range of auto-immune conditions) and pre-clinical development work on its VSEL™ technology. The Company's most advanced asset is AMR-001for the treatment of acute myocardial infarction for which enrollment for a Phase II study in the United States commenced in January. AMR-001 is administered 5 to 11 days post-stent placement in patients diagnosed with an ST segment elevation myocardial infarction ("STEMI") with ejection fraction less than or equal to 48%. The manufacturing, product supply, and logistics for the trial will be supported by Progenitor Cell Therapy, LLC, NeoStem's contract manufacturing company.

StemCells, Inc. (STEM), with an MCAP of $21.8 million, reported its 2011 update on March 13th. StemCells reported a net loss of $21.3 million or $1.50 per share, compared to a net loss of $25.2 million, or $2.05 per share, for 2010. Total revenue in 2011 was $1.2 million compared to total revenue of $1.4 million for 2010. Other income in 2011 was $6.8 million relative to $4.1 million for 2010.

The following pertinent 2011 stem cell research highlights were noted:

  1. In February 2011, the fourth and final patient in our Phase I clinical trial in Pelizaeus-Merzbacher Disease, was enrolled and transplanted with our proprietary HuCNS-SC(R) cells (purified human neural stem cells). This trial is the first to evaluate neural stem cells as a potential treatment for a myelination disorder.
  2. In March 2011, we initiated a Phase I/II clinical trial of our HuCNS-SC human neural stem cells in chronic spinal cord injury.
  3. In September 2011, the first patient in our Phase I/II clinical trial in chronic spinal cord injury was enrolled and successfully transplanted with our HuCNS-SC cells.
  4. In December 2011, we successfully completed the enrollment and dosing of the first cohort of patients in our Phase I/II clinical trial in chronic spinal cord injury.
  5. In January 2012, the U.S. Food and Drug Administration (FDA) authorized the initiation of a Phase I/II clinical trial of our HuCNS-SC cells in dry AMD, the most common form of AMD.

Aastrom Biosciences, with an MCAP of $96.6 million, reported its 2011 update on March 12th. Aastrom reported a net loss of $19.7 million, or $0.51 per share, for 2011 compared to a net loss of $25.5 million, or $0.90 per share, for 2010. As of December 31, 2011, the company had a total of $5.5 million in cash and cash equivalents, relative to $31.2 million in cash and cash equivalents on December 31, 2010.

The following pertinent 2011 stem cell research highlights were noted:

  1. Initiated patient enrollment in the Phase III REVIVE-CLI clinical study of ixmyelocel-T in patients with critical limb ischemia.
  2. Reported and presented positive 12-month results from the Phase IIb RESTORE-CLI clinical study at the American Heart Association Scientific Sessions.
  3. Received a notice of issuance of a key composition-of-matter patent from the European Patent Office for ixmyelocel-T.
  4. The company also noted "we now have the momentum and financial resources to advance our clinical programs in critical limb ischemia and dilated cardiomyopathy and pursue productive discussions with a number of potential partners this year".

Nueralstem Inc. (NYSEMKT:CUR), with an MCAP of $55 million, announced its 2011 review on March 29th. The company announced a net loss for 2011 of $12.5 million ($0.26 per share) relative to a net loss of $18.4 million ($0.42 per share) for 2010. Net cash used in operating activities decreased to $8 million in 2011 from $9.98 million in 2010. Cash and cash equivalents on December 31st were $2.35 million relative to $9.26 million for the same period in 2010. The company had $5.7 million in cash as of March 8, 2012.

The following pertinent 2011 stem cell research highlights were noted:

  1. We also received notices of allowance for our proprietary surgical spinal platform and floating spinal cannula as well as the method for delivering a therapeutic agent to a spinal cord target. We believe this breakthrough surgical device, invented by our ALS surgeon, Dr. Nicholas M. Boulis, and for which Neuralstem holds the exclusive worldwide license, will be the industry standard for such intraspinal procedures.
  2. In February 2011, Neuralstem received FDA orphan drug designation for the treatment of ALS with its human spinal cord derived neural stem cells (NSI-566RSC)
  3. The first patient of the final six cervical transplantation Phase I ALS patients received the world's first neural stem cell injections in the gray matter of the upper spinal cord region in November 2011, at Emory University. It was reported that the entire 18-patient trial will conclude six months after the final surgery
  4. Neuralstem was selected as the principal subcontractor under a U.S. Department of Defense contract to develop its human neural stem cell technology for the treatment of cancerous brain tumors ... The contract award was $1.6 million for the first year of the project, of which Neuralstem received $625,000. In this new approach to oncology, it was reported that the neural stem cells will be engineered to attack brain cancer in three ways: by expressing an antibody known to suppress tumor growth; by expressing an enzyme that selectively kills tumor cells; and by expressing an antiangiogenic protein that will starve the tumors by preventing the formation of the blood vessels that feed them.

Athersys, Inc. (NASDAQ:ATHX), with an MCAP of $40.6 million, reported its 2011 review on March 27th. For year ending December 31sth, the company reported revenues of $10.3 million relative to $8.9 million for 2010. Net loss was reported at $13.7 for the year relative to $11.4 for 2010. Cash and equivalents for year end 2011 totaled $12.8 million relative to $15.2 million for 2010.

The following pertinent 2011 stem cell research highlights were noted:

  1. Announced positive results from Phase I clinical trial evaluating MultiStem® treatment for individuals undergoing allogeneic hematopoietic stem cell transplants (HSCTs) for the treatment of leukemia and related conditions and the risk for graft-versus-host disease.
  2. Published in Circulation Research the results from a successful Phase I clinical study of MultiStem treatment of patients who had recently suffered an acute myocardial infarction.
  3. Granted a patent covering the use of non-embryonic multipotent stem cells for the treatment of cardiovascular conditions, capping a productive year for our stem cell patent prosecution.

Author's Comments:

The stem cell sector, like all biotechs or small pharmaceuticals, is a volatile and risky venture for investors. Most of the sector is construed as "development Phase" with no marketable products to generate revenue via sales or licensing. As these companies grow, progress through clinicals, and advance by trial and error during various stages of the clinicals, much data is generated and much knowledge is gained. These are expensive ventures requiring much company capital and time. While most of these biotechs rely on shareholder contributions and larger pharmaceutical partnerships, these can detract from the existing shareholders' value via stock dilution and sharing of potential earnings, respectively.

NeoStem and Aastrom, both with recent financing behind them, present shareholders the unique opportunity of investment in stem cell companies with current manufacturing capabilities. NeoStem's advantage over Aastrom is particularly appealing as its manufacturing capabilities not only benefit the company directly as a direct source of its stem cell therapies, but the company has also been supplying well-known companies such as ImmunoCellular Therapeutics and Baxter International through revenue-generating manufacturing contracts. Although not contributing enough to enable the company to generate a profit at this time, the revenue does serve the purpose of off-setting some of the clinicals costs and also improves the company's networking abilities and exposure to potential future suitors. In a sector struggling to blossom and yield fruit, the early stage stem cell companies generating revenue and networking with larger pharmaceuticals can offer investors something to consider as they look to diversify their biotech holdings. 2012 and beyond could prove to be exciting and hopefully profitable times for stem cell biotechs and those investors fortunate enough to gain entry-level positions into one of biotech's most exciting sectors.

Disclosure: I am long NBS.

Continue to Part II >>