Cell Therapy Industry HiLites 2009-06-12

“Autologous manufacturing is not the deal breaker it used to be. If these [cell-based] vaccines work for really difficult-to-treat diseases, the manufacturing issues will be worked out.” Howard Liang, MBA, Ph.D., Analyst and Biotechnology Managing Direct at Leerink Swan. "Special Report: Customized Cancer Vaccines Finally (Maybe) Arrive". GEN News Highlights. 12 June 2009.

Amen. Glad to hear someone else saying it.

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FINANCIAL

Neuralstem, Inc (NYSE AMEX: CUR) received notice from the NYSE Amex Exchange that its shareholder’s equity has dropped below the $2,000,000 threshold required by the NYSE Amex for continued listing. The Company intends to submit a plan to the Exchange. CEO and President Richard Garr said, “We are confident that the company will be in compliance.”

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National Institutes of Health has awarded a $5 million grant to Q Therapeutics (co-founded by former NIH researcher Mahendra Rao), the University of Utah's Cell Therapy Facility and Johns Hopkins University School of Medicine to start human clinical trials of Q-Cells for myotrophic lateral sclerosis, or ALS -- better known as Lou Gehrig's disease.

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Opexa Therapeutics, Inc. (NASDAQ: OPXA) received a favorable ruling from NASDAQ allowing an extension to regain compliance and continue the listing of the Company’s securities on The NASDAQ Capital Market.


CLINICAL

StemCells, Inc. (NASDAQ: STEM) announced positive data from its Phase I clinical trial of its proprietary HuCNS-SC® product candidate (fetal-derived purified human neural stem cells). The six-patient Phase I trial was designed primarily to assess the safety associated with transplanting HuCNS-SC cells as a treatment for infantile and late infantile NCL, often referred to as Batten disease

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Celsense, Inc. is one of the recipients in a 4-year $1.95M grant from the National Institutes of Health to test its in vivo cell trafficking MRI reagent in human clinical studies. The funded studies will be the first effort to evaluate Cell Sense, the Company’s fluorocarbon-based magnetic resonance imaging (MRI) cell tracking reagent, for clinical use. Additionally, the studies will garner preliminary data critically important for improving immunotherapeutic delivery strategies in metastatic colorectal (CRC) and other forms of cancer.

The Cell Sense technology makes transplanted cells visible by MRI. Therapeutic cells are labeled ex vivo with the Cell Sense reagent prior to administration to the patient. Cell migration and biodistribution are subsequently monitored using fluorine-19 (19F) MRI or magnetic resonance spectroscopy (MRS).

The key advantage of Cell Sense is purported to be that the 19F images are extremely selective for the labeled cells, with no background signal from the host’s tissues. Furthermore, accurate cell quantification in regions of interest is possible. Existing data from pre-clinical studies using human cells is said to show that the Cell Sense reagent is highly biocompatible inside cells and does not affect their innate function.

A clinical-grade version of the Cell Sense reagent has been formulated by Celsense, Inc. and is currently being subjected to mandatory in vitro and animal safety studies in preparation for human trials.

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Never a company to shy away from making bold and early claims, Advanced Cell Technology, Inc. (OTC:ACTC.PK) Chairman and CEO William M. Caldwell IV said earlier this week that they are completing the "finishing touches" on a submission to seek FDA approval to conduct human clinical trials with a cell therapy that may be able to address many of the 200 plus known retinal diseases.

COMMERCIAL

It's hard to put this story under any one category but since the financial terms weren't released and it's not yet clinical, I'll call it "commercial". J&J's Ortho Biotech Oncology Research & Development, a unit of Centocor Research & Development, Inc., has announced that it has entered into a five-year Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), with Steven A. Rosenberg, M.D., Ph.D., chief, Surgery Branch, serving as the NCI principal investigator, to research and develop novel cell therapy technologies as potential treatments for a variety of cancers.

The deal bring together Ortho's and Rosenberg's adoptive immunotherapy technologies designed to work by helping the immune system fight cancer.

Rosenberg has been a pioneer in the field of adoptive immunotherapy of cancer developing Tumor Infiltrating Lymphocytes (TILs) - T cells obtained from a patient's tumor, expanded and then re-administered to actively seek and destroy cancer cells. In recent years, Dr. Rosenberg's team developed a new technology in which T cells obtained from a patient's blood are genetically engineered to express receptors that give them specific immunity against cancer cells and then re-administered.

Meanwhile, researchers at Ortho Biotech Oncology Research & Development independently developed a different and proprietary adoptive immunotherapeutic approach that uses tumor antigens and other materials to stimulate T cells from a patient's blood to become Cytotoxic T Lymphocytes (CTLs), which recognize and attack tumor cells.

Both technologies show postive early clinical results in melanoma, a type of skin cancer that ranks sixth among U.S. men and seventh in U.S. women for the most commonly diagnosed cancer, according to the NCI.

Under the CRADA, Dr. Rosenberg's lab will conduct a clinical trial in melanoma patients using Ortho Biotech Oncology Research & Development's proprietary technology. It is hoped that the technology will be effective in other types of cancer, as well.

They expect to begin clinical testing of the immunotherapy technology in melanoma patients by the end of 2009, with the possibility of additional studies for other types of cancer and other technologies in years to come.

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Cellular Dynamics International, Inc. has secured an exclusive license on a patent portfolio surrounding cardiovascular progenitor cells. The license covers differentiation of stem cells into all cell types of the human heart. The patent portfolio was generated by and obtained from Gordon Keller, Ph.D. and Mount Sinai School of Medicine.

Cardiovascular progenitor cells are capable of further differentiation into the multiple cell types of the heart, including cardiomyocytes, endothelial cells, and vascular smooth muscle cells, both in vitro and in vivo. CDI intends to commercialize the technology to the pharma and biotech industry for predictive toxicology and screening purposes. Iit's not clear whether the license also includes clinical applications but if so they may be looking for licensing partners given their current lack of interest in developing these cells into therapeutics.

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In a not-so-surprising move, StemCells Inc (NASDAQ: STEM) has decided to close StemCell Sciences in Melbourne, Australia after its acquisition of the company in March. British staff will now be holding their breath to see if the same will happen to them. STEM says it has offered relocation packages to employees should they wish to move to sunny California. This leaves one "stem cell" firm in Australia where previously there were four (SCS, Bresagen, Embryonic Stemcells Inc and Mesoblast). If there is good news it is that, if I recall correctly, the three others were acquired and not just shuttered.

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After many months of actively looking to offload its contract manufacturing subsidiary, Fresenius Biotech, has finally sold EUFETS AG to BioNTech AG. BioNTech is looking ot leverage the acquisition to further its quest to develop innovative molecular immunotherapies and biomarker based diagnostic approaches for individualized treatment of cancer and other severe human diseases.

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Having used a contract manufacturer for what and when they needed, BioLife Solutions has now reverted back to internal manfucturing with the completion of construction and validation of their internal GMP manufacturing facility. Recently they gleefully released the first production lot for sale of their biopreservation media products made in the new Bothell, WA plant.

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StemCyte Therapeutics is collaborating with Chennai-based Apollo Hospitals in joint venture with Ahmedabad-based Cadila Pharmaceuticals to set up what reports to be a first-of-its-kind umbilical cord blood (UCB) public bank for India.

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Ok this news is a little soft but its a product that continues to be reinvented and a company we don't hear from much so I'll give them the space. HepaLife Technologies, Inc. (OTCBB: HPLF) (FWB: HL1) (WKN: 500625) has expanded their Advisory Board to support planning (e.g., refine the clinical strategy and patient treatment protocols) for their expected bioartificial liver clinical trial. HepaLife is developing a cell-based bioartificial liver system, HepaMate, as a potentially lifesaving treatment for liver failure patients. HepaMate is comprised of a blood plasma separation cartridge, a hollow-fiber bioreactor filled with proprietary porcine liver cells, a charcoal column, an oxygenator, circuit tubing and a plasma reservoir. These components are assembled into a patented blood/plasma circulation system, which is placed on the HepaDrive perfusion platform. The HepaMate technology has previously been tested in clinical studies involving more than 200 patients and has been the subject of over 50 scientific papers. It is anticipated that the upcoming trial would be a pivotal trial of the technology.

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ThermoGenesis Corp. (Nasdaq: KOOL) mentioned in a recent press release that their Res-Q system - a point-of-care system designed to collect, process, and reinfuse bone marrow stem cells for therapeutic use, is anticipated to be launched on the market this month. Presumably in markets outside the US? They also discussed that they had received $500,000 in an SBIR grant from the NIH to study and develop biomaterials that can be used to deploy placental stem cells in bone repair and regenerative medicine applications. Now free money is free money but this seems a bit of a stretch to me for a company not without its struggles in its penetrating the market with its current cord blood storage and cord blood/bone marrow process systems. Apparently the the Res-Q platform is also being studied for potential use in the preparation of platelet rich plasma (PRP) from peripheral blood. Give them credit for continuing to think long-term and cross-platform despite struggles with their existing products.

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"No more pads or tampons, period." Lifecell International is set to introduce in India next month menstrual cups as a cost-effective option to control menstrual flow in women and as a preparatory move for the company's menstrual-blood banking project called Femme, which is scheduled to begin in July


MISCELLANIA

Xconomy's Luke Timmerman does a nicewriting up an informative piece on Denderon's recent AGM and the thinking around the company of what will be involved in taking the company "all the way".

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Ok its another pre-clinical item (which I rarely cover) but again this one's just too cool to ignore. The New Scientists did a nice job of looking at Doris Taylor's work at University of Minnesota. Her team is looking to expand on their success creating rat hearts using stem cells. The ultimate goal is to some day create a limitless supply of transplant organs for humans. The team took stripped-down scaffolding of one rat heart and coated it with tissue produced from another rat's stem cells. They now want to repeat the procedure using hearts and other organs from human cadavers or larger animals.

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The NIH received 49,015 comments in response to its draft guidelines for human stem cell research. One of those was submitted by CIRM.

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President Barack Obama should appoint a commission on stem cell research to help the therapy fulfill its promise to save lives and reduce medical costs, says Geron Corp. Chief Executive Officer Tom Okarma.

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The California Institute for Regenerative Medicine (CIRM) received 73 preapplications for the CIRM Disease Team Awards RFA 09-01. The principal investigator for 15 of those applications is based with a for-profit entity. All of the teams proposed a wide variety of collaborators with several of the not-for-profit principal investigators citing collaborators in the for-profit sector. After review by a panel of outside experts and the CIRM in-house science team, a subset of these teams have been asked to prepare a full application, which will be due to CIRM July 16. Word on the street is that to-date just over 20 of the 73 have been given the green light to the next step.

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StemCellDigest.net has posted a handy chart covering the public companies they follow in the stem cell sector showing the types of stem cells and business from which theyy hope to derive income as evidenced by the company's own statements, owned intellectual property, and/or areas of research. The one critically useful but missing column is the phase in which each of these products in.

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One of my contacts, Scott Shields, Reimbursement and Health Policy Consultant at Argenta Reimbursement Advisors and owner of Blue Train Consulting Group, LLC has launched "The Reimbursement Wiki".

It promises to be a valuable online resource for reimbursement information particularly for those of us who do not work in that field full time. Below is his announcement of the site's launch:

I am announcing the public availability of my Reimbursement Wiki. I have compiled links to coding, coverage, and payment information, as well as information such as the following: - Medicare (URLs for rules, topics such as surgeries, compounding, CED, MS-DRGs, DME, inpatient list, laboratory and diagnostic testing, etc.) - Managed Care (URLs for medical/coverage policy sites at various payers, technology assessment organizations, literature on coverage decision making, etc.) - Medicaid (URLs for CPT/HCPCS code lookup by state for payment info, state fee schedules, drug product data, etc.) - Payment (URLs for Medicare payment basics, top 200 CPT/HCPCS codes, top 100 lab procedures, literature on drug pricing, etc.) There is plenty more -- basically, reimbursement and health resources I have collected in one place for reference purposes. The URL is http://reimbursement.atwiki.com. The Reimbursement Wiki is ongoing project of mine, and I add material based on topics of current work, so not all topics are represented equally. Nevertheless, I find it an invaluable resource, and I hope you do, too. Access to the Reimbursement Wiki is free. Please register at http://reimbursement.atwiki.com/signup, and email me your @ID. I will then invite you through the wiki. My email address is scott@argentaadvisors.com, or you can contact me via LinkedIn.

Sign-off

Organogenesis, Inc has established an annual scholarship program with Canton High School to honor a graduating senior pursuing a career in life sciences research or biomedical engineering. Kudos for them. We need more of that! Me? I sponsored a child this week. No big deal but it reminds me to remind you...

...find a way to make a difference in your world today.

One more thing. You want to make sure you hear cell therapy-related news faster? Get on Twitter and follow me. I'm @celltherapy.

Ok. Hope you found that useful. I'm trying to get back to my every Friday schedule here. I'll do my best. Happy weekend.

Lee Buckler, BEd, LLB
Cell Therapy Group
Principal

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Cell Therapy Group. Your Cell Therapy Business Consultancy.
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Another imminent M&A in cell therapy?

Is another company with commercial cell therapy products about to be bought for a song?

York Pharma PLC (AIM:YRK) is a pharmaceutical Group established in 2003 specializing in the acquisition, development and commercialization of novel prescription dermatological products to market and supply branded prescription products to pharmaceutical wholesalers, hospitals and general practitioners within the field of dermatology.

University of Sheffield spin-out CellTran Ltd was incorporated in 2000 specializing in developing cell therapies to heal wounds. During its existence the company succeeded in bringing two cell therapy products into the UK market for the treatment of chronic wounds and burns: Myskin™ and Cryoskin™. CellTran majority shareholders were Biofusion plc, White Rose Technology Limited, The YFM Group, South Yorkshire Investment Fund, Partnerships UK and the Wellcome Trust.

In my December 12 post here on this blog, I reported the following:

One of the more important bits of news I'm late to the table for (announced at the end of October) is the acquisition of "certain of the assets and the continuing business of Celltran Limited (“Celltran”) from its administrators" by York Pharma plc (AIM: YRK) for what it describes - in the very undestated British way - a "compelling valuation".

This seems an apt description when one learns that despite the fact that "approximately £15 million has been invested in the Celltran portfolio of products" to-date, York bought the company's two commercial product plus "a rich pipeline of associated products and technologies" and the ongling business for - make sure you're sitting down - "£70,000 plus the payment of royalties on future sales for a period of five years (at the greater of £100,000 (£20,000 per year) or 10% of future revenues generated by the products over such period)".

The real reason this is such a compelling valuation is that Celltran had - and now York Pharma has - two cell therapies on the market (albeit only in the UK) in Myskin™ and Cryoskin™.

But the news does not end there. York also announced simultaneous to the acquisition that they had completed an institutional placing raising £510,000 (before expenses) to provide "additional working capital for the integration, operation and further development of the assets within York Pharma’s business." York, established in 2003, describes itself as a pharmaceutical group with particular focus on dermatological products which makes the two cell therapy products fit well in their existing portfolio.

Now York Pharma is on the ropes.

It was reported today that "shares in the AIM-listed York Pharma were suspended from trading after the company said that it still hadn’t managed to secure any short term funding and as a result would miss the March 31 deadline to publish its annual results. Earlier this month York said it was in talks with two potential offerors and that those discussions included the provision of short term funding facilities. The company said it needed the cash to allow it to continue trading through the period needed to complete any formal offer."

It was then reported later today that "one of the potential offerors has today entered into an agreement with the Company to provide the Company with a secured revolving Credit facility of up to US$1,000,000" and that "drawdown of the full facility should allow the company to continue trading through the period needed to complete any formal offer".

Now this begs the question: who is the suitor? One one hand this smacks of exactly the kind of move StemCells Inc has made more than once in the past. Would this fit with their other recent UK acquisition? With two products already on the market would this address STEM's need for immediate revenue to fund its ongoing therapeutic development programs? While the answers might be "yes", I doubt this kind of move is within STEM's bandwidth given the ink is not yet even dry on the SCS deal (not sure it is even closed). If not STEM, then who?

Certainly this would be a fit with Advanced BioHealing's portfolio or that of Karocell Tissue Engineering AB, FIDIA Advanced Biopolymers, or Forticell Bioscience (formerly Ortec International, Inc.). Would Genzyme or a pharma company pick this up? I have my doubts. Perhaps an Asian company like Japan Tissue Engineering Co., Ltd. (J-TEC)? It might also be quite compelling to merge this with Intercytex and create a company with several commercial cell therapy products and a rich pipeline of products.

It's likely none of the above but it is fun to speculate. I'm sure we'll find out in the next few days...

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.

Cell Therapy HiLites 2008-11-14

Here's what caught my attention this week:

Celgene received FDA clearance to proceed with its placenta-derived stem cell clinical ttudy - a phase I, multi-center trial for moderate-to-severe Crohn's disease.

MiscroIslet filed for Chapter 11 bankruptcy protection but expects to restructure as a private company, continue operations, and file its first IND by year's end for an encapsulated porcine-islet product to treat diabetes.

Celladon released promising phase I results for the first gene therapy trial for advanced heart failure.

Cytori is starts to sell some of its StemSource® cell banking systems for collecting, processing, and storing adipose-derived cells. The company received its first order from Asian distributor Green Hospital Supply. The cell bank is to be set up in Japan. Despite all the continued skepticism about the value of cell banking to the donor-potential-patient, cell banking businesses are popping up prolifically around the world on the promise of short-term revenue to fund long-term R&D while providing a service some believe to be of irreplaceable value.

Although technically not a company-related cell therapy story, one of the more interesting developments this week was the announcement out of Charite in Berlin that they have a patient who is AIDS-free following a unique stem cell transplant. The patient developed leukemia which the docs were going to treat with a stem cell transplant. But rather than do the routine transplant by finding a match and treating just his leukemia they dared to be a little different. They went looking for a donor that matched and also had a CCR5-gene mutation (a naturally-occurring mutation which makes life difficult for the HIV virus according to some studies). The Berlin team found 80 compatible bone marrow donors for their patient, an unusually high sum, two of whom had the CCR5-Gene mutation. One of these they used with surprising and heartening results. What I like about this story almost as much as the happy ending and exciting discovery is how the doctors are trying to control the hype around this single-patient data and early results. It's not likely that this kind of transplant is a future treatmtent (for reasons related to supply, demand, and other commercial realities) but perhaps there is a kernal of a cell therapy product here somewhere that someone as innovative as these doctors will develop in time.

Ok I'm late on the draw here but it just came to my attention that in August NovaRx initiated a pivotal phase III in lung cancer patients. Watch on this blog in the coming days for more on the perception-vs-reality gap related to the number of industry-sponsored cell and gene therapy trials currently active worldwide.

Introgen may disappear while on the brink of success. Despite have an MAA being reviewed in the EU for their gene therapy for head and neck cancer, their BLA submission for the same therapy was rejected by the FDA as incomplete (currently under appeal). The company is now "exploring options" after a troubled Q3.

While not for its cell therapy program, Athersys announced it had achieved a clinical development milestone in its Bristol-Myers Squibb alliance. The amount of the milestone payment has not yet been revealed but any revenue is good revenue to offset the cell therapy development burn.

Bucking recent trends, StemCells Inc. (STEM) announced it will raise $20M in a stock sale to select institutional investors. Good news for any company in these times. Even better news for the cell therapy sector!

Finally, in the most over-reported news of the day, Pfizer opens up a regenerative medicine shop. I agree this is big news. This is not a minor investment; it's not throwing some money at a portfolio of biotechs in hopes that something will stick; it's not using stem cells as tools for drug testing or discovery. This is evidence of a belief by those in the halls of some of the tallest towers in life sciences, that cell therapies are going to change the way we treat people. Here are a couple quotes:

“Pfizer Regenerative Medicine represents a great opportunity to focus world-class research in a field that holds considerable promise for biomedical science and for the treatment of many debilitating conditions such as diabetes and neurodegenerative disorders.” said Rod MacKenzie, PhD, head of Worldwide Research at Pfizer.

The unit will be led by Chief Scientific Officer, Ruth McKernan, PhD, who said “I’m very excited to lead this new research unit. While there is still a lot to understand about how stem cells can be used therapeutically, we believe it is one of the most promising areas of scientific research.”

Ok, one more. Corey Goodman, PhD, President of Pfizer’s Biotherapeutics and Bioinnovation Center, said, “The formation of this new unit represents another key step forward in Pfizer’s commitment to be at the forefront of new approaches in biotherapeutics and bioinnovation and to expand our research efforts and expertise into emerging areas of biomedical science, like regenerative medicine, that have great potential for human health.”

Apparently the unit will operate independently (in the fashion of smaller biotechs) with about 70 researchers when fully staffed - in both Cambridge, UK and Cambridge, Mass - and work collaboratively with "leading academic, biotech and pharmaceutical partners around the world.


That's the cell therapy industry as I saw it this week. Happy weekend.