Cell Therapy Industry HiLites 2009-03-06

I have silly and serious news. Apparently within just one hour after taking a capsule of the "wonder supplement" from the bottle pictured to the right, 3-4 million stem cells will be added to my bloodstream and go to work repairing and regenerating my body! That's the silly part. The serious part is that I'm told they made $1 million in the first month of sales.

This week was marked by another clinical hold (sorry I can't tell you who), a release from clinical hold (Aastrom), some significant M&A, and my inability to keep pace with the pace of revolutionary changes in new ways to make iPS cells - ways which appear safer and hold more promise for the eventual therapeutic potential of these cells. I have to confess to not really getting on the iPS bandwagon until last week when I heard some great talks and really just spent some time trying to understand what all the fuss was about. As usual, Monya Baker has put together a nice summary of this week's IPS progress.

For those of you who haven't seen it yet, this year's TED talk by Juan Enriquez (Beyond the crisis, mindboggling science and the arrival of Homo evolutis) is a fascinating insight into what he cites are three 'evolving' trends that will change society as we know it and should be considered when looking beyond the current financial crisis. The ability to engineer cell-based therapies is one of them.

And now, for the news and analysis ...

FINANCIAL

The big news of the week was StemCells Inc. (STEM) announcement that it has agreed to acquire the operating subsidiaries and assets of Stem Cell Sciences PLC (STEM.LN) for 2.65 million StemCells Inc. shares. At its closing price of $1.56 per share Feb. 27, the deal values Stem Cell Sciences at $4.85 million. Shareholders representing 30% of Stem Cell Sciences have backed the deal. "The industrial logic of this acquisition is compelling," said Martin McGlynn, president and chief executive of StemCells. StemCells' expertise is with stem cells for therapeutic uses, while Stem Cell Sciences has focused on non-therapeutic applications like assays for drug discovery. For more details see my post from earlier this week.

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Athersys, Inc. (Nasdaq:ATHX) today announced it will host its first annual Investor Day being held at the Intercontinental Hotel on Wednesday, April 8th, 2009 in New York City. The meeting will begin promptly at 8:30 a.m. Eastern Time and end at approximately 11:30 a.m. Eastern Time. Institutional investors and analysts interested in attending should contact Lisa Wilson, Investor Relations for Athersys: details available at the company website.

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Hon Hai Precision Industry Co has invested in local cell-tissue banking and cell therapy company Bionet Corp (TPO:1784) through the purchase of NT$100 million (US$2.86 million) in Bionet shares. The purchased shares represent 8.6 percent of Bionet’s outstanding shares. The two companies plan to invest an additional NT$200 million in a joint venture called Conn Lian, aimed at developing next-generation health care focused on predictive, preventive, personalized and participatory medicine. Hon Hai will take 50 percent ownership of the newly formed company, while Bionet and the rest of the investment consortium will each take 22.5 percent and 27.5 percent. Bionet is engaged in the storage and application of stem cells, as well as gene detection. Under the name BabyBanks, the company provides stem cell banking from cord blood, umbilical cord, placenta, teeth, and mobilized peripheral blood stem cell storage in mutiple Asian countries.

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Adam Feuerstein, the irreverent biotech analyst at TheStreet, has identified 10 key upcoming events he says will play a leading role in determining how well biotech stocks perform as we move from a cold winter to spring. Among his top picks for market moving events? Phase III data on Dendreon's(NASDAQ: DNDN) Provenge expected to be released within the next 60 days.

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Advanced Cell Technology, Inc. (OTC: ACTC) continues, once again, to provide evidence of its veritable immortality, with an an announcement that it has received a total of $400,000 in additional funding through NIH research grants and the receipt of the final payment in connection with its recently formed international joint venture with CHA Biotech Co, Ltd., its Korean-based biotechnology company focused on the development of stem cell technologies. Proceeds from these transactions as well as other transactions in advanced discussions will be used to support the Company's retinal pigment epithelium cells (RPE) program. The Company anticipates filing an IND with the FDA during the second half of this year.

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Bioheart, Inc. (Nasdaq:BHRT) got a notice of delisting from the NASDAQ Capital Market. The Company is in the process of engaging a market maker for its common stock and causing the required application to be filed for quotation of the Company's common stock on the OTC Bulletin Board. Howard Leonhardt, Chief Executive Officer of Bioheart, commented: "It is unfortunate that Bioheart's stock price has been so adversely affected by the general decline in the securities markets that it could not maintain its NASDAQ listing. The Company has been current in its filings with the SEC and there are many good things happening at Bioheart with regard to the commercialization and sales of its products that we expect will add value to the company's equity. We look forward to re-establishing a robust market for our shares and having them approved for quotation on the OTC Bulletin Board."

Of course IguanaBio's couldn't help but take a lighthearted look at the unfortunate news...

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Isolagen, Inc. (AMEX:ILE) currently estimates that its unrestricted, available cash resources will allow the Company to continue in operation for approximately three weeks. The Company continues to pursue potential financing alternatives and potential strategic partnership discussions. As previously disclosed, the Company currently has a debt liability of approximately $89.7 million related to its 3.5% subordinated notes, which could be called due, at the option of the note holders, as early as November 2009. Interest on the notes is due semiannually on May 1 and November 1. The Company is pursuing the potential sale of its 57% ownership interest in Agera Laboratories, Inc.

In the meantime, Isolagen is reporting positive clinical news and is on brink of filing a BLA.

CLINICAL

Isolagen, Inc. (AMEX:ILE) announced positive top-line efficacy results from a Phase II/III clinical study (Study IT-A-008) of the Isolagen Therapy for the treatment of moderate to severe acne scars. The study met all primary efficacy endpoints and was statistically significant, making this a major clinical milestone for the Company. The Phase II/III, placebo-controlled study investigating the efficacy and safety of Isolagen Therapy for the treatment of moderate to severe acne scars evaluated a total of 109 people at seven clinical sites across the United States. In the study, both the Patient and Evaluator assessments met the co-primary endpoints and were statistically significant.

The Company believes that to ultimately obtain FDA approval with respect to the acne scar indication, the Company will require FDA concurrence with the use of the Company's Evaluator Live Acne Scar Assessment scale, which the Company developed specifically for use in this study and which has not been previously used in a clinical trial. Further, if the Company obtains this concurrence from the FDA, then the Company will also require, at a minimum, one additional Phase III study to complement study IT-A-008 in order to pursue FDA approval and licensure.

The production process for the Isolagen Therapy is a proprietary cellular processing system that creates a natural, living cell therapy. By multiplying a person's own collagen-producing cells, or fibroblasts, into tens of millions of new cells, a personalized treatment is created that is then returned to the person's skin. Isolagen Therapy, is designed to improve skin damage caused by the normal effects of aging, sun damage, acne and burns.

The Company is preparing to submit its Biologics License Application (BLA) for Isolagen Therapy for the treatment of wrinkles/nasolabial folds and currently expects this submission to occur within two weeks.

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Cytori Therapeutics, Inc. (NASDAQ: CYTX) completed enrollment in its APOLLO trial to evaluate the safety and feasibility of adipose tissue-derived stem and regenerative cells, processed with the Company’s Celution® System, in the treatment of severe heart attacks. According to the company, the steering committee deemed, in concurrence with data safety and monitoring board (DSMB), that the safety and feasibility goal in APOLLO was met in thirst cohort of 12 patients at the initial cell dose and that the procedure did not raise safety concerns. Cytori has said they will report results on the primary six month follow up period for the APOLLO trial in late 2009.

“... we needed to show that the fat harvest could be done safely on sick heart attack patients and that we could re-deliver the stem and regenerative cells back to these patients through the coronary artery in the same surgical procedure early in the peri-myocardial infarction period", said Marc H. Hedrick, M.D., president, Cytori Therapeutics.

The steering committee has recommended that a pivotal study be conducted to further evaluate efficacy. Cytori is presently in discussion with its notified body in Europe to negotiate study size needed to show efficacy and achieve reimbursement.

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Aastrom Biosciences, Inc. (Nasdaq:ASTM) reported that the U.S. Food and Drug Administration (FDA) has removed the clinical hold from the Company's U.S. Phase II IMPACT-DCM clinical trial and that patient enrollment would resume at the four initiated clinical sites. The IMPACT-DCM trial is evaluating the use of Cardiac Repair Cells (CRCs), a mixture of stem and progenitor cells derived from a patient's own bone marrow, for the treatment of dilated cardiomyopathy (DCM), a severe form of chronic heart failure. George W. Dunbar, President and Chief Executive Officer at Aastrom has said that the company still anticipates completing patient enrollment in this trial by the end of calendar year 2009.

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Pluristem Therapeutics Inc. (NasdaqCM:PSTI)(DAX:PJT) announced that the US Food & Drug Administration (FDA) has cleared the Company's Investigational New Drug (IND) application to initiate a Phase I clinical trial for the treatment of Critical Limb Ischemia (CLI), the end stage of peripheral artery disease (PAD), using Pluristem's PLX-PAD. In this Phase I dose ranging trial, to be conducted at multiple locations in the US, PLX-PAD will be administered to patients considered "late stage" and defined as patients afflicted with CLI that have not responded to traditional medical or surgical interventions.

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Opexa Therapeutics, Inc. (NASDAQ:OPXA) announced that continuing analysis of the data^® for Early Relapsing Multiple Sclerosis) for the treatment of MS, has shown that Tovaxin® Multiple Sclerosis therapy shows significant improvement in visual impairment. An analysis of the MS Quality of Life Inventory Data (MSQLI) from the 150 patient study has shown that in the complete modified intent to treat (mITT) population (n=142), patients treated with Tovaxin demonstrated a statistically significant improvement in the Impact of Visual Impairment Scale scores (p=0.028) compared to those on placebo. This improvement was observed within six months of completing the full course of treatment. Visual problems are a common, often disabling symptom in MS and according to the National Multiple Sclerosis Society can affect over 40% of patients. The Impact of Visual Impairment Scale consists of 5 items that assess the extent to which various activities dependent upon vision are affected by MS-related visual problems.

The company is now on life-support levels of cash and continues evaluate a number of strategic opportunities and advance partnering discussions for both their T-cell technology in MS and stem cell technology in diabetes.

COMMERCIAL

Pluristem Therapeutics Inc. (NasdaqCM: PSTI) (DAX: PJT) announced it has signed a collaboration agreement with Pharmicell Europe GmbH. Under the Agreement, Pharmicell will distribute PLX-PAD, Pluristem’s placental-derived adherent stromal cell product, to various clinical sites in Germany, in connection with Pluristem’s upcoming clinical trials for the indication of Critical Limb Ischemia. Pharmicell will distribute PLX-PAD to the clinical sites at Charité-Universitätsmedizin, and Franziskus-Krankenhaus, both located in Berlin. “PLX-PAD should be stored in a location close to the clinical sites in Berlin, so it could reach the patients in a timely manner when treatment is required,” stated Zami Aberman, Chairman, President and CEO of Pluristem. “Collaborating with Pharmicell gives Pluristem the advantage of storing this ready-to-use cell product under stringent conditions in a German regulated facility prior to dispensing it for patient use,” he added.

Pharmicell Europe GmbH was founded in Berlin in 2006 with the goal of providing consumers with new adult stem cell therapies. Having completed preclinical trials for stroke and myocarditis with Charité and BCRT, in 2009 Pharmicell intends to commence clinical trials using fat-derived cells for plastic and aesthetic therapy for European customers. Pharmicell aims to open a specialized stem cell treatment center. As the European subsidiary of FCB Pharmicell Co. Ltd., based in South Korea, Pharmicell Europe’s R&D laboratories are located in the Center of Berlin and in the Europarc Dreilinden in Brandenburg which hosts Pharmicell’s GMP certified cell production area.

Pharmicell Europe GmbH is not to be confused with Pharmacell BV, the Dutch-based cell theapy contract manufacturer. Formed in 2005, Pharmacell is a contract services company which also has Asian connections. In 2006, CyGenics Ltd. (ASX: CYN), a cell therapy, tools, and services company with operations many of the countries in Asia, bought what was then a 20% stake in Pharmacell. The investment came with an option - which to the best of my knowledge, if still alive, has not been exercised - to purchase the remaining 80% at a later date. Since then the company attracted €2 million in an early 2007 financing from other sources.

Then late last year, Tokuda Hospital Sofia and the Maastricht-based PharmaCell announced an agreement to establish a joint-venture to build and operate a GMP facility in Sofia, Bulgaria. Tokuda Hospital Sofia is owned by the Tokushukai Medical Corporation, one of the largest health institutions in the world with over 280 health care facilities in Japan; among which 65 hospitals. Tokushukai Medical Corporation is expanding its activities outside Japan. The Tokuda Hospital Sofia is the first of these expansions and is reportedly one of the most modern hospital in Eastern Europe. The joint-venture will build a 'world-class" GMP facility focussed on human cell & tissue culturing in the Tokuda Hospital. From there it intends to serve the whole Eastern European Region for these treatments. The total investment in the Sofia Facility is reported to be €5 million and construction was scheduled to start in early 2009.

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Israeli firm, Piercell, has announced a collaboration with immuno, cell and gene therapy expert, Dr. Cyril Cohen. Piercell aims to integrate its innovative biomolecular delivery device - a robust transfection solution for primary and hard to transfect cells - with cell therapy protocols such as Dr. Cohen's.

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Privately-held Stemedica Cell Technologies, Inc. as been on a very public company and profile-building campaign since the beginning of the year. In January, they announced the appointment of Dr Nabil Dib, MD as Special Advisor to its Board of Directors and the appointment of Michael Bayer, MD, as Director, Medical Services, responsible for developing pre-clinical and clinical trial protocols. In February, they announced the appointment of Riccardo Nisato, MBA, PhD, as Director of Manufacturing and Clinical Business Development of Stemedica International, CH. Dr. Nisato will be responsible for replicating Stemedica's U.S. based operations to Stemedica's International's center based in Lausanne, Switzerland. February also saw the announcment that they had filed with the Food and Drug Branch of the State of California's Department of Public Health for a California Drug Manufacturing License for adult stem cells for U.S.-based Phase I and Phase II clinical trials at the their facility in San Diego, California. The company rounded out the month publishing a commentary on the PLoS Medicine article published online February 17, 2009 describing a patient who developed “brain tumors” (glioneuronal neoplasms) after undergoing repeated transplants of “fetal neural stem cells” in Russia starting in 2001.

This first week of March saw Stemedica announce that the leadership of the California Institute for Regenerative Medicine (CIRM) had recently completed a two part visit of their manufacturing facility, R&D lab and corporate headquarters. It's not entirely clear to me why CIRM has blessed Stemedica with not just one but two visits when to the best of my knowledge it has not nor does it have any plans to visit the other two corporate contract manufacturers in California.

Furthermore, CIRMS's President, Alan Trounson is quoted as saying: "I was glad to have the opportunity to visit Stemedica and get to know more about its leadership, plans for the future and to see first hand the assets and potential they represent to the stem cell movement in California. We want to have strong working relationships with all sectors of California's stem cell industry, including for-profit companies like Stemedica."

The praise from CIRM didn't stop there. Dr. Marie Csete, CIRM's Chief Scientific Officer is quoted as saying, "We're delighted to see Stemedica make the effort to secure a clinical grade manufacturing license from the State of California. We believe this capability will aid numerous organizations in California and around the world in their efforts to manufacture stem cell products for clinical application."

Just to round out what sounds like a veritable love-in, Stuart Lipton, M.D., Ph.D., Scientific Director and Senior Vice President of the Del E. Webb Center for Neuroscience, Aging and Stem Cell Research at the Burnham Institute for Medical Research, San Diego, CA, who joined Dr. Trounson during his visit to Stemedica's manufacturing facility provides the following statement of affection: "I was impressed with the progress that Stemedica has made on the licensing process for clinical grade allogeneic stem cell manufacturing and am excited about expanding our research relationship with the Stemedica team. Having a manufacturing resource like this in California will be a great asset to our entire research community. I anticipate that many CIRM-funded stem cell research institutions will take advantage of Stemedica's manufacturing facility that will soon become FDB (Food & Drug Board) cGMP licensed."

It's hard to imagine that CIRM could provide a more overt and public statement of support for a company which will likely be, if it has not already, vying for its funds as one of several contract service providers in California. This is a curious position for a granting agency like CIRM and certainly carries with it the risk of the perception of preferential treatment or bias. I will certainly be following this up with CIRM.

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Off-shore stem cell clinics continue to gather steam. Local news coverage from Texas is an example of the kind of media attention being given these companies every day by telling the stories of dozens of American patient testimonials for these off-shore healers. Today's example is the Institute for Cellular Medicine ("ICM") operating under the name Cell Medicine out of San Jose, Costa Rica. They are treating Autoimmune Diseases, Cerebral Palsy, Critical Limb Ischemia, Degenerative Joint Disease, Diabetes Type II, Heart Failure, Multiple Sclerosis, Osteoarthritis, Rheumatoid Arthritis and Spinal Injury with an autologous concoction for stem cells.

ICM is, in fact, a licensee of Medistem Inc. (OTC BB:MEDS.OB). The two entities were consolidated reporting companies until December, 2007 when for various financial and commercial reasons it was seen beneficial to seperate the entities somewhat though ICM is controlled by the Company’s Chairman and majority stockholder.

Last year MediStem also entered into a licensing agreement with a third party for the exclusive use of certain Medistem technologies and know-how in the countries of India, Malaysia, Pakistan, Bangladesh, Sri Lanka, Nepal, Maldives, Bhutan, Afghanistan, Indonesia and Thailand. In exchange for the grant of the exclusive license, the Company received cash, a non-controlling equity interest in the licensee and a license to use and commercialize all of the third parties’ current and future stem cell technologies. They also, I believe have a similar licensee in Mexico.

Medistem Inc., formerly Medistem Laboratories, Inc, is a U.S. based biotechnology company focused on the development and commercialization of adult stem cell-based technologies used in the treatment of inflammatory and degenerative diseases. Their primary focus is commercialization of their "endometrial regenerative cell” (“ERC”) sourced from menstrual blood. These cells originate in the endometrium where they are believed to have one vital function: to make new blood vessels (angiogenesis). Stimulation of this process is believed to offer hope for patients with circulatory disorders in which certain tissues are lacking oxygen because of restricted blood flow. As reported here, Medistem filed its first IND in January for a phase I trial of its ERC cells for use in the treatment of critical limb ischemia.

RESOURCES & EVENTS

Pittsburgh Symposium on Molecular & Cellular Imaging. April 3-4, 2009.
Carnegie Mellon University

Molecular and Cellular Imaging enable the visualization of cellular trafficking, cellular function, and molecular processes in living organisms. In the coming years, these rapidly developing disciplines will provide an avenue to translate basic biomedical science to clinical breakthroughs for numerous diseases and cancers, as well as a means to monitor drug targeting and cellular therapies. Carnegie Mellon University and the University of Pittsburgh will host a 1-½ day symposium on Cellular and Molecular Imaging to showcase current cutting-edge research in areas of Magnetic Resonance Imaging (MRI), Positron Emision Tomography (PET), Optical Microscopy, and Ultrasound.

Registration is free, but space is limited.

Faculty includes:
Joseph J. H. Ackerman, Washington University at St. Louis
Eric T. Ahrens, Carnegie Mellon University
Ronald Blasberg, Memorial Sloan-Kettering Cancer Center
Jeffrey W. M. Bulte, Johns Hopkins University
Christopher H. Contag, Stanford University
Alan P. Koretsky, National Institutes of Health
Martin G. Pomper, John Hopkins University
Stephen H. Thorne, University of Pittsburgh

Stanford's Office of Technology Licensing (OTL) has just released technologies that may be of interest. The technologies are titled: Multipotent Neural Stem Cells Derived From Human Embryonic Stem Cell.

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International Stem Cell Corporation Hosts Webinar, "Stem Cells 102: Beyond Embryonic Stem Cell Research," Thursday, March 12, 2009, From 1:00pm-2:00pm EDT

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International Stem Cell Corporation (OTCBB:ISCO), whose primary technology is human "parthenogenetic" stem cells from unfertilized eggs, will host a webinar titled "Stem Cells 102: Beyond Embryonic Stem Cell Research," Thursday, March 12, 2009, from 1:00pm-2:00pm EDT. This free educational webinar is for journalists, shareholders, and investment professionals interested in learning about new emerging technology in the stem cell research arena. Please note that space is limited. Interested parties are invited to make a reservation to participate in the webinar by using this link.

Covered in the webinar: What is a pluripotent stem cell? What are the implications of the FDA's recent approval of Geron clinical trials? What is parthenogenesis and how does this process solve certain ethical and immune rejection issues that have long shadowed stem cell research? How close are we to treating diseases with stem cells?

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Dr. Kiki Sanford, the hula hooping, TKD black-belt wearing, radio show producing physiologist- now-science-journalist, blogger and 'sexy geek' at The Birds Brain has posted a little inaptly named but nontheless useful, easy-to-read-for-the layperson-summary of the history of iPS cells entitled "A Brief History of Stem Cells".

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ISCT would like to ask for your assistance with completing a survey on quality plan implementation. The results will be presented at the 2009 ISCT Annual Meeting.

Sign-off

So...here I am once again a little off my Friday target but don't you dare complain!

That's all the business news and analysis I could muster. I think the news this week certainly reflects the times. We had companies with money scooping up companies without it, we have companies on the brink of commercial products that are struggling to raise enough money to get to the finish line, more paradigm-shifting scientific breakthroughs, some signs of significant investment still proceeding outside the U.S. and a few more shenanigans on the periphery of the sector that we hope don't malign the industry by association.

That, my friends, is a wrap on my insights into the cell therapy biz for this week.