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Tuesday, March 31, 2009

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...

Monday, March 30, 2009

Cell types available for regenerative medicine applications


A useful teaching image and an article that's recommended reading:



Cell types available for regenerative medicine applications.
Click on image to view larger version.

Excerpted from: Stem cell bioprocessing: fundamentals and principles

Madsrk R Placzek, I-Ming Chung, Hugo M Macedo, Siti Ismail, Teresa Mortera Blanco, Mayasari Lim, Jae Min Cha, Iliana Fauzi, Yunyi Kang, David C.L Yeo, Chi Yip Joan Ma, Julia M Polak, Nicki Panoskaltsis† and Athanasios Mantalaris*

† Biological Systems Engineering Laboratory, Centre for Process Systems Engineering, Department of Chemical Engineering, Imperial College London
South Kensington Campus, London SW7 2AZ, UK

*Author for correspondence (a.mantalaris@imperial.ac.uk)

doi:
10.1098/​rsif.2008.0442 J. R. Soc. Interface 6 March 2009 vol. 6 no. 32 209-232

Abstract

In recent years, the potential of stem cell research for tissue engineering-based therapies and regenerative medicine clinical applications has become well established. In 2006, Chung pioneered the first entire organ transplant using adult stem cells and a scaffold for clinical evaluation. With this a new milestone was achieved, with seven patients with myelomeningocele receiving stem cell-derived bladder transplants resulting in substantial improvements in their quality of life. While a bladder is a relatively simple organ, the breakthrough highlights the incredible benefits that can be gained from the cross-disciplinary nature of tissue engineering and regenerative medicine (TERM) that encompasses stem cell research and stem cell bioprocessing.

Unquestionably, the development of bioprocess technologies for the transfer of the current laboratory-based practice of stem cell tissue culture to the clinic as therapeutics necessitates the application of engineering principles and practices to achieve control, reproducibility, automation, validation and safety of the process and the product. The successful translation will require contributions from fundamental research (from developmental biology to the ‘omics’ technologies and advances in immunology) and from existing industrial practice (biologics), especially on automation, quality assurance and regulation.

The timely development, integration and execution of various components will be critical—failures of the past (such as in the commercialization of skin equivalents) on marketing, pricing, production and advertising should not be repeated. This review aims to address the principles required for successful stem cell bioprocessing so that they can be applied deftly to clinical applications.


Friday, March 27, 2009

Cell Therapy Industry HiLites 2009-03-27


Here's a big thank you shout out to the growing cadre of great readers, subscribers, and participants in the network of cell therapy colleagues I'm proud to be part of whether it is here on this blog or on our LinkedIn Cell Therapy Industry Group. We're proving cell therapy means business! Keep up the outstanding repartee.




In keeping with the biblical proposition that "nothing new is under the sun", a new physician group as been formed to "oppose FDA's position on adult stem cells". The newly formed "American Stem Cell Therapy Association (ASCTA)" this week posted its manifesto online and issued a press release saying the "organization was formed in response to the Food and Drug Administration's (FDA) recent position that the adult stem cells found in everyone's body are drugs, a position the ASCTA opposes." I'm not sure which move the FDA made recently that would make these docs think this is the FDA's recent position. As I wrote on this blog back in September last year, doctors have been trying to tell the FDA that cell therapy is the "practice of medicine" for years and it hasn't worked. Of course, it will come as no surprise to you that central to this new movement of doctors lobbying for the right to treat patients with their own stem cells however they see fit, is Dr. Chris Centeno of Regenexx and other doctors like Dr. Zannos Grekos who are involved with stem cell treatment clinics marketing to US patients for clinics performing their magic outside the USA.


In a classic case of overstating the point, ASCTA member Dr. Frank Falco states, "The FDA's position against someone using their own stem cells is taking it too far." Of course, saying that is the FDA's position, is taking his point too far but subtleties like that don't get people engaged in a revolution!

Enough of that. On to the news & analysis. It wasn't a great week for cell therapy with Osiris stopping its phase III trial for Chron's and all but that had more to do with the difficulties of designing good clinical trials than it did cell therapy.

FINANCIAL

Intercytex Group Plc (AIM: ICX) announced it is in talks which may lead to an offer for the company. In February, the company said it was reviewing options, including a possible sale or merger, after it stopped work on Cyzact, one of its main products, to preserve cash. The company's product Vavelta has reportedly now treated 120 people in a commercial setting and its ICX-SKN skin graft replacement for burns and acute wounds is fully funded by the US Armed Forces Institute of Regenerative Medicine. Inercytex reported revenues of £17,000 for the year.

*
Taiwan is angling to be one of the major biotech hubs of Asia by making biotechnology the country's third major industry in the next 10 years. To that end, it has proposed a $1.76 billion dollar VC fund. The Taiwan government's National Development Fund will have a 40 percent stake in the venture, and the private sector will see to the other 60 percent. The economic development plan also calls for the establishment of a biotech incubation center that will introduce new medicines to existing biotech parks. Taiwan has a strong interest in cell therapies and the country's regulatory authority(the BFDA) is investing heavily in establishing a well-defined regulatory framework for cell, gene,and tissue based products. Unlike Singapore's investment in stem cell research, Taiwan appears to be positioning itself to support later-stage commercial entities already working on translation of research into clinical products.

CLINICAL

And here's the big news of the week. Osiris Therapeutics, Inc. (NASDAQ: OSIR) announced it was stopping its phase III clinical trial of Prochymal for the treatment of acute Crohn’s disease (CD).

The good news? It was not for safety concerns. The decision was made after the trial's final scheduled interim analysis showed that one of the two Prochymal dose arms (they don't know which because that has not yet been unblinded) in one of the two trials in the program had crossed a futility boundary (would not achieve statistical significance) according to the study's DSMB.

Osiris’ phase III CD program consisted of 2 trials, and induction trial (S-603) and a maintenance trial (S-610). The Study-603 “Induction” trial was randomized into three arms, one-high dose Prochymal (400M cells for the 1st two infusions and 200M cells for the next two) , one low-dose Prochymal (200M cells in the 1st two infusions and 100M cells in the next two) and placebo. After 28 days of dosing, patients were evaluated by a subjective evaluation for a reduction in the Crohn’s disease activity index (CDAI). Patients with a 100-point drop in CDAI score according to the self-evaluation were then eligible for re-randomization into the longer-term Study-610 “Maintenance” trial. It is this design that management now believes presented an inducement for patients to "over-report" improvements so as to be eligible to participate in the longer, subsequent trial. This is evidenced, they believe, by the fact that 56% of the participants in the in the S-603 program enrolled in the S-610 program when they would have expected the number to be more around 30 - 40%.

The other pieces of good news to salvage out of the day are (a) Genzyme supports the decision, and (b) the trial had already enrolled 210 of its expected 270 patients. Osiris is going to complete the study as if it were a 210-patient study in hopes that there is significant data from the trial that can not only be used in redesigning subsequent trials but also to bolster Prochymal’s overall safety database. Regarding Genzyme's position, this is certainly reason for them to be disappointed but this saves them milestone payouts in the short-term and they are not on the hook for the additional costs that will be incurred because of the decision so as long as they still believe in the fundamentals of the product, they have no reason to pull out of the relationship now.

Randy Mills spent some time in a webcast on Friday explaining the details of what transpired, the decision, what they theorize went wrong or was wrong with the trial, and where they anticipate going from here. He made it very clear a number of times that there is no reason to believe this will have any impact on the Prochymal trial for GvHD. One of the other things he explained was that Osiris never believed this current phase III program for Crohn's was going to be sufficient to support a BLA. They had always anticipated another phase III trial. What Randy didn't say was whether this decision would mean another one or two trials would be required.

Analyst Jason Napodano is on record stating he believes "it could be a year or so before Osiris can re-initiate the Crohn’s program, which will most likely include two separate phase III trials, one induction and one maintenance, but once initiated the program should enroll quickly given management’s experience from just halted program and the inclusion of several additional new centers that did not participate the first time." Despite the setback he believe the news presents a buying opportunity for Osiris shares which were down as much as 22% in Friday's trading.

He believes "Osiris remains financially sound and should exit 2009 with over $100 million on the books" and expects "Prochymal will be on the market in the U.S. by the end of next year for GvHD". They have a target of $25 per share.

In Friday's investor teleconference, Randy stated he expects the company will be reporting top-line data on the Prochymal trial for steroid-refractory GvHD in 3Q 2009 given that enrollment in that trial is already complete and, based on enrollment rates for the acute GvHD trial, he expects to be reporting data from that trial in the same Quarter.

*
Neurotech Pharmaceuticals, Inc. announced that the Company's lead product candidate, NT-501, substantially slowed the loss of vision in a Phase 2 clinical trial in subjects with dry age-related macular degeneration involving geographic atrophy. NT-501 is an intraocular implant that consists of human cells that have been genetically modified to secrete ciliary neurotrophic factor (CNTF) which is delivered directly to the back of the eye in a controlled, continuous basis by means of the Company's proprietary Encapsulated Cell Technology platform, thereby bypassing the blood-retinal barrier. The Phase 2 study is a multi-centered, randomized, double-masked, sham-controlled study of 51 subjects with GA. Patients received either a high or low dose NT-501 implant or a sham treatment in one eye only. The high dose of NT-501 stabilized best corrected visual acuity at 12-months, with 96.3% (p=0.078) of treated-patients losing fewer than three lines of vision, or 15 letters, versus 75% of the patients in the sham-treatment group.

What I find interesting about the study is that five devices from this trial have now been explanted 12 months following implantation and all have been found to have uniformly healthy, viable cells that continue to produce therapeutic levels of CNTF. This is reportedly consistent with data from multiple trials of NT-501 in which, to date, 23 devices have been explanted between 12 and 18 months following implantation and all devices have contained healthy, viable CNTF-producing cells.

The clear implication is that the implanted devices would continue to excrete therapeutic levels of CNTF longer than the 1-year threshold for the study. This is proof-of-principle for Neurotech's Encapsulated Cell Technology platform which may well have sundry other applications.

*
Will Dendreon's unblinding and announcement of interim analysis data for Provenge back in October be its final bad decision? Four top statisticians say Dendreon may have compromised the integrity of the trial by putting out the release. They say it was unorthodox for Dendreon to even know such a detailed result, much less to publicize it. The danger: The company, patients or doctors might have changed what they were doing once they knew how the study was going. If the final outcome is only marginally statistically significant, it might be tossed, putting Dendreon and its drug back at square one. The statisticians are left scratching their heads at the data release. "I have no idea what their rationale would have been," says Susan Ellenberg, a statistician at the University of Pennsylvania. "I can't rule out the possibility that they did have a reason I'd be comfortable with, but I can't think what it might be."

*
Finally there is some news about a US company treating patients in a clinic outside the US in a way that many would say has hallmarks of scientific credibility. Two years ago DaVinci Biosciences, headquartered in Costa Mesa, California, treated 52 acute and chronic spinal cord injury patients in Ecuador with injections of their own bone marrow-derived stem cells. They conducted follow-up studies and have now published the results for the first 8 patients in issue 17(12) of Cell Transplantation. The follow-up report claims that MRIs have revealed "noticeable morphological changes within the spinal cord after administration of autologous bone marrow derived stem cells." There was no tumor formation, increased pain or deterioration of function following administration of the stem cell treatment. The researchers conclude that the therapy proved safe and effective in improving their quality of life. Although there are plenty of stem cells clinics claiming anecdotal evidence (not published in peer-reviewed journals) of the therapeutic effect of such treatments and there have been numerous studies in animals demonstrating the benefits of stem cell treatment for the treatment of spinal cord injury, this may be the first published study of its kind.

COMMERCIAL

In what is now becoming a trend between large pharma and research institutes, the Salk Institute announced a strategic alliance agreement with Sanofi-Aventis establishing the Sanofi-Aventis Regenerative Medicine Program (SARP). Financial terms of the three- to five-year agreement were not revealed.

Saying that the program was without "restrictive preconditions", the announcement was vague on details about the anticipated nature of the collaboration other than it would sponsor "institute-wide discovery grants in promising research areas that address the organizations’ mutual interests" It was also unclear what types of results or products Sanofi expected to get from the program other than "research retreats and "extended working lab visits". The San Diego Business Journal reports that Sanofi-Aventis will have the option to license any discoveries that result from the collaboration. It's not clear to me how this fits with the deal Salk made last year when it partnered with another French pharmaceutical company, Ipsen, in a deal worth $10 million over five years. It's also not clear to me if this is an investment in cell as therapies or more about cells as tools. We can not necessarily infer the former simply from the "Regenerative Medicine" name put to the program.

*
Stratatech Corp. announced that it has launched the StrataTest® human skin model. Composed of both an epidermis and a dermis, the StrataTest® human skin model is said to display the physical, chemical and histological characteristics of native human skin. The tissue is supplied in a 24-well format for consumer product testing, drug discovery and toxicity screening.The StrataTest® human skin model, which is intended for research use only, is manufactured using Stratatech’s proprietary NIKS® human keratinocytes. Stratetech believes the product offers a "superior, cost-effective, in vitro testing skin model that it believe enables better prediction of in vivo biological response for consumer product, drug discovery and other toxicity testing applications.”

*
Stepping into a space owned primarily by MaxCyte and Lonza's Amaxa BioSystems, Invitrogen, a division of Life Technologies (NASDAQ:LIFE) announced the launch of it's "Neon Transfection Device", a bench-top device for the delivery of DNA, RNA, and proteins into a wide range of mammalian cell types, especially difficult to transfect cells, such as many types of primary and stem cells. The Neon Transfection Device is reportedly well suited for gene and siRNA delivery into stem cells, features a unique transfection chamber that minimizes cell death, has minimal reagent requirement, and works with many different cell types.

*
Progenitor Cell Therapy, LLC (PCT) announced that Lisa Doria-Cavuoto will be joining the company as Vice President of Commercial Cell Therapies, effective April 1, 2009. In this role, she will be responsible for managing the day-to-day business operations of PCT's commercial stem cell processing, storage, and clinical distribution service.

*
Former GE Healthcare & Thermogenesis executive, Dan Segal, is heading up a newly minted private cord blood bank in Orange County, California called PacificCord.

RESOURCES, EVENTS, & MISCELLANEA

ISCT has released a call for Late Breaking Abstracts in the following categories:
  • Mesenchymal and Tissue Stem Cells
  • Hematopoietic Stem Cells
  • Gene Therapy
  • Immunotherapy and Dendritic Cells
  • Cell and Tissue Evaluation
  • Lab Practices
  • Legal and Regulatory Affairs
  • Translational Process Development
Deadline for Submissions: April 3, 2009. Notification of Abstract Status: April 10, 2009

Click here to submit.

Sign-off...

I spoke this week to an American-trained plastic surgeon now practicing in Asia. In 2006 he paid (presumably handsomely) to attend a symposium hosted by 3 European "professors" on commercial "cell therapy" techniques that are being sold today in select jurisdictions and which could bring longevity and youthfulness to clients everywhere and, I assume, fame and fortune to the doctors brave enough to sell them. One was "live stem cell therapy" using rabbit fetal cells and the other was "fresh/frozen cell therapy" using cells from sheep embryos (or placenta?). He was so impressed he bought into the program, tried it on himself, his family, and then, convinced it was safe, started to sell it to clients. The good doctor I spoke with was using the sheep product, as many others are doing, and administering them not topically but intra-muscularly for "skin rejuvenation".

Long story short, he is now being prosecuted by his Medical Council for providing non-evidenced based medicines. Here's the rub. This all sound quite ghastly to many of us but there is at least one clinic in Switzerland that has been injecting sheep cells into people for over 50 years without any apparent safety issues and to the endless, anecdotal accolades of clients who claim enhanced youthful visages, energy, sex drive, longevity, etc. What's more, there is an internet site where you can buy sheep placenta capsules and even human placenta injectibles. I won't link to any of these sites because I don't want to give them the web traffic but a quick Google search will lead you to multiple clinics and distributors.

I don't envy the position of regulators. As much as a clear and enforceable regulatory framework is critical to the industry, so is a strong and properly financed regulatory authority. Equally important is that we as an industry be vigilant in protecting the quality of our science, our medicines, and our patient's health. We are not grappling with easy issues here. Perhaps the injection of sheep or rabbit fetal cells are the cure-all they are reported to be but what it they're not and we're injecting them into human for non-life-threatening conditions?

...

That's the kind of week its been. Feel better about yourself and tell a friend about the Cell Therapy Blog today! :-)


Wednesday, March 25, 2009

Cell Therapy Industry HiLites 2009-03-25

I am more intrigued every day by the potential for identifying opportunities in the convergence of cell therapies, diagnostics, personalized medicines, theragnostics, regenerative medicine, and more traditional biotechnologies and pharmaceuticals.

I wonder to what extent we will look back on these primitive days of ex vivo cell therapy and chuckle at its ephemeral but educational existence much like betavision or pervasive therapeutic bloodletting. I suspect many of these therapies will have a role in future medicine but I'm starting to believe more and more that the future of cell therapy will be more about
in vivo manipulations than ex vivo manufacturing.



In an attempt to catch up from my brief hiatus and to have more frequent and less tome-like posts, I bring you this mid-week edition of Cell Therapy Industry HiLites.

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Contact Lee [at] celltherapygroup [dot] com
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FINANCIAL

Given the continued extraordinary market conditions, NASDAQ has further suspended enforcement of the rules requiring a minimum $1.00 per share. closing bid price and a minimum market value of publicly held shares until Monday, July 20, 2009. Good new for many including Aastrom Biosciences, Inc. (Nasdaq:ASTM) which now has until September 18, 2009 to regain compliance with the $1.00 minimum closing bid price rule in order to remain listed on the Nasdaq Capital Market.

As promised, International Stem Cell Corporation (OTCBB:ISCO) has received the third $1 million tranche of an anticipated private equity financing of up to $5 million the remainder of which is says will be funded over the next several months.

Now that Cell Genesys, Inc. (NASDAQ:CEGE) has no products to develop things are looking up for it! The company reported financial results for the fourth quarter and full year ended December 31, 2008. The company reported a net loss of $13.3 million ($0.15 per fully diluted share), for the fourth quarter of 2008, compared to a net loss of $33.4 million ($0.43 per fully diluted share) for the corresponding period in 2007. The net loss for the year ended December 31, 2008 was $47.0 million ($0.56 per fully diluted share), compared to a net loss of $99.3 million ($1.39 per fully diluted share) for the year ended December 31, 2007. As of December 31, 2008, the Company had $86.1 million in cash, cash equivalents and short-term investments. The Company currently expects to have approximately $73 million and $69 million in cash, cash equivalents and short-term investments at March 31, 2009 and June 30, 2009, respectively. The Company continues to explore strategic alternatives, including merger with or acquisition by another company, further restructuring, allocation of its resources toward other biopharmaceutical product areas, and sale of the Company’s assets and liquidation of the Company.

CLINICAL

As a result of ongoing and active discussions in our LinkedIn Cell Therapy Industry Group, it has come to my attention thatprivately-held Harvest Technologies Corp - a company known for its bone marrow and platelet concentrate devices - has entered into the therapeutics business. It recently obtained an Investigational Device Exemption (IDE) from the FDA to conduct a randomized, controlled, double-blind, multicenter clinical trial using the Company’s BMAC System to prepare a composition of bone marrow stemcells to treat patients with Critical Limb Ischemia (CLI). This “feasibility trial” will enroll a total of 48 subjects. Subjects who enroll in this study will have to have exhausted all surgical and procedural options and are at extreme risk for major amputation. The BMAC System is a point-of-care device for concentrating a patient’s own (autologous) bone marrow stem cells in approximately 15 minutes at the bedside. The clinical study design provides for injecting these cells into the affected limb to reduce the potential for limb amputation. It also will offer data with respect to the safety of this procedure.

What's even more interesting is that reportedly the company has submitted an investigation device exemption for a double-blind placebo cardiac safety study—to inject BMAC cells into the myocardium during bypass surgery in patients with severely compromised heart function.

This presents an interesting regulatory pathway for a couple of business models both in providing point-of-care devices and providing therapeutic service employing such devices. More on that in an upcoming blog (and in our LinkedIn discussions).

The Indian Council of Medical Research and the Drug Controller General of India is said to have now approved protocols Bangalore-based firm Stempeutics Research for multi-center phase I/II double blind randomized clinical trials of bone marrow derived ex vivo cultured adult mesenchymal stem cells for Elevated Acute Myocardial Infarction and Critical Limb Ischemia. The cells are to be administered by injection.

COMMERCIAL

MultiCell Technologies, Inc. (OTC Bulletin Board: MCET - News) announced it has entered into a cooperative research and development agreement with Maxim Biotech, Inc. to develop products for the study of liver stem cells and liver cancer. The cooperative research and development agreement with Maxim Biotech, Inc. will initially focus on the development of a family of life science research reagent tool kits which can be used to isolate liver stem cells, and help to elucidate liver stem cell gene function and their encoded proteins. MultiCell plans to further leverage this research effort involving liver stem cells to identify therapeutic targets, and diagnostic and prognostic markers of liver cancer. MultiCell will also seek to develop and patent therapeutic product opportunities specifically targeting the treatment of primary liver cancer and intrahepatic bile duct cancer.

Kosdaq-listed RNL Bio (famous for dog cloning) faces a KFDA ban on its cosmetic containing human-derived stem cells.

J&J's Therakos, Inc. announced the U.S. Food and Drug Administration (FDA) approval of the THERAKOS™ CELLEX™ Photopheresis System for the palliative (reducing the severity of symptoms) treatment of the skin manifestations (appearance) of cutaneous T-cell lymphoma (CTCL) that are unresponsive to other forms of treatment. The THERAKOS™ CELLEX™ Photopheresis System is a system that uses extracorporeal (outside the body) photopheresis (ECP) cell therapy to relieve the symptoms of CTCL. The system also has been cleared recently in Canada and Europe. The new THERAKOS™ CELLEX™ Photopheresis System is said to feature several improvements over but retain the primary benefits of the predecessor THERAKOS™ UVAR™ XTS™ Photopheresis System. The improvementa are said to open up this treatment option to patients for whom it was previously unfeasible including lower weight patients.

Stem Cell Therapy International, Inc. (OTCBB: SCII) decided it was worthwhile to announce that they are in "strategic meetings" with unnamed "global stem cell representatives" and they are "not wasting any time...with the merger of Stem Cell Therapy International, Inc. and Histostem, Ltd. of South Korea" to create the newly name company, AmStem International. Now that's great news.

StemCelDigest.net outlined the single-digit degrees of similarity between companies (STEM, ISCO, ACT) pursuing stem cell solutions for diseases of the retina. Other companies in this space include the Pfizer-backed EyeCyte and UK's ReNeuron.

RESOURCES & EVENTS

Watch for an announcement from ISCT announcing a call for late-breaking abstracts.

Jon Rowley (Lonza) reports that there is more stem cell content - from science to manufacturing - at BIO2009 this year. Dare we think it's not a bubble this time?


Sign-off

A proposal to change the name of the American Society of Gene Therapy (ASGT) to the American Society of Gene and Cell Therapy (ASGCT) has been nanimously approved by the Board of Directors and the proposal is now being forwarded to the general membership of the Society for a final vote in April of 2009. In an editorial in December issue of Molecular Therapy, Membership Committee Chair and Past ASGT President, Savio LC Woo, stated that the Membership Committee has recommended that the following concrete steps,among others, be implemented to maximize its positive impact.
  • First, the term “cell therapy” must be included in the Society’s mission statement.
  • Second, the Society’s leadership should contact the leadership of the International Society for Cellular Therapy (ISCT) to assure them that our name change represents an opportunity for both societies to collaborate and advance our common interests and to initiate a dialogue with them onthe needs and interests of cell therapists so as to better recruit these researchers into our society.
  • Third, the Society leadership should reach out to leading investigators and the research communities in adult and ES cell biology as well as iPS cell biology.
  • Fourth, the Program Committee should ensure that cell therapy is richly represented in the symposium programs of the Society’s annual meetings.
  • Fifth, the Nominating Committee will need to make a concerted effort to identify and nominate prominent cell therapists into the Society’s leadership positions.
  • Finally, the editorial team of Molecular Therapy—the Society’s official journal—is encouraged to increase even further the important efforts they have made over the past few years to recruit Editorial Board members with expertise in cell therapy and stem cell engineering, in additionto continuing their successful solicitation of outstanding articles in these areas.
Professional organizations, it seems to me, are facing the collective pressures of increasing financial constraints, the rise of online access to publications minimizing the need for member-rate subscriptions, and the networking power of online social networks, all combining to make their value proposition much less obvious to many - particularly the younger set.

I understand ASGT's desire to ensure its mandate stays relevant and inclusive of all aspects of therapies employing genes. I also believe the increasing overlap between organizations representing "cell therapy" is not in the best interests of the sector. While ISCT remains the only organization with cell therapy of all types at the core of its mission, AABB has encroached into the cell therapy space from the blood banking side, ISSCR from the basic stem cell research end of the spectrum, ASBMT from the clinical stem cell transplant side, TERMIS from the tissue enginering, and now ASGT (following the precedent set by ESGT-ESGCT) from the gene therapy end.

As C. Mason & P. Dunhill wrote last year in Regenerative Medicine, what the industry needs is a strong voice generated by a powerful industry association. As long as we continue to create more silos of representation, we will continue to stray farther away from what's needed. While I have strong ties to ISCT and continue to serve that organization in several capacities, I am increasingly frustrated by the dilution of this nascent industry's time, money, and efforts spread over too many organizations and conferences without creating the voice for industry that is needed.

I'm not sure where this frustration will take me but my increasing conviction is that the companies in the cell therapy sector are ill-represented in any organization and spattering attempts at representation throughout all of them (including BIO which has to-date all but ignored the sector) is increasingly insufficient.

Am I off base here? Is this a real need or am I making something out of nothing? What do you think?


Tuesday, March 17, 2009

Cell Therapy Industry HiLites 2009-03-13

I know, I know. I'm way off schedule. I'll try to post some shorter, snappier editions of the Cell Therapy Industry HiLites over the next few days in order to catch up but you know what they say about good intentions...



CIRM is repositioning itself more firmly as a translational agency, preparing for NIH to take on some of the burden of funding basic research.

One cell therapy and one cell-based gene therapy made R&D Direction list of 100 great investigational drugs for 2009 as selected by the editors: Prochymal (allogeneic mesenchymal stromal cells) by Osiris/Genzyme for acute mycardial infarction and VRX496 (
lentiviral vector transduced autologous CD4 T cells) for HIV/AIDS.

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Cell Therapy Blog wants your ad. If you're looking to get high-impact and lasting exposure to those in the cell therapy industry...


Contact Lee [at] celltherapygroup [dot] com
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FINANCIAL

Perhaps a sign of the times, I discovered this posted on LinkedIn recently:

Opportunity to acquire assets of a preclinical diabetes therapy company in Chapter 7

MicroIslet, Inc. has developed a treatment for Type 1 Diabetes involving the transplantation of non-human islet cells. Due to the company's previous capital structure and the collapse of the capital markets in late 2008, MicroIslet filed for Chapter 11 reorganization. The bankruptcy case was converted to Chapter 7 liquidation last month.


The estate owns significant intellectual property and is party to an exclusive contract to source islet cells from a unique supplier. Management estimates a six month time frame to file the IND. Animal and toxicity studies are complete with favorable results.

For more information about the company and its assets visit www.microislet.com or reply to me.

Thank you
Brian Conn
Chief Financial Officer at MicroIslet, Inc.

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Last year, Cytori Therapeutics, Inc. (NASDAQ:CYTX) raised $12 Million in February in an equity purchase and strategic collaboration agreement with Green Hospital Supply, then in August they announced raising $17 million from a private placement financing led by Olympus Corporation (TSE: 7733) with participation from select institutional investors, and finally in October they entered into a $15 million loan facility with GE Healthcare Financial Services and Silicon Valley Bank.

Despite all this, in the company's Form-10K filed with the SEC on March 6, 2009, the Company's auditors issued an audit opinion that includes a matter of emphasis paragraph relating to the Company's ability to continue as a going concern.

Consquently, this week the company announced it had closed and received net proceeds to raise $10 million, before placement agent fees and offering expenses. The funds will be used for sales and marketing activities related to the commercialization of the Celution(R) System and consumables as well as other related and complementary products, for ongoing clinical studies of the Celution(R) System for breast reconstruction and cardiovascular disease, for ongoing research and development to support the Company's products and its pipeline development, and for general working capital.

Due to the closing of the financing subsequent to the issuance of the auditor’s opinion, Cytori has substantially improved its cash position, which the Company believes
may fund its operations through at least 2009 as described in the liquidity and capital resources discussion of its Form-10K.

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MolMed S.p.A. (Milan:MLM) Board of Directors reviewed and approved the draft year-end financial statements as at 31 December 2008. The company reported a Positive net financial position of €35.3 million, consisting of cash, cash equivalents and current financial assets, operating costs totalling € 23.1 million, and a loss of € 17.4 million (an average of over €1.4 million/month on average), as compared to a loss of € 12.7 million in 2007. The notable improvement of MolMed’s net financial position, from € 5.7 million as at 31 December 2007 to € 35.3 million as at 31 December 2008, is due to the IPO proceeds of €56.2 million derived from MolMed’s listing at the Milan Stock Exchange.

Almost in passing the year-end review stated that MolMed and
Takara Bio Inc. have agreed to terminate their research collaboration in AIDS gene therapy to give full focus to their respective, more advanced programs .

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Stem Cell Therapy International Inc. (OTC BB:SCII.OB) announced that they have obtained short term financing, enabling the Company to "update" and finalize the merger between Stem Cell Therapy International, Inc. and the Histostem Corporation, Ltd. (Korea) which has been filed previously with the SEC. I don't know what it means to 'update' a merger but it would appear this loan will get it finalized.

The Company got a whopping $150,000 and 6 months or less to repay to the lenders $187,500.
This short term financing will allow the Company to continue to "pay necessary vendors to maintain the Company's public filing requirements, begin collaborations of operations between Histostem and AmStem International, finish the documents necessary to continue with the private placement and proceed with the execution of the Company's strategic plan."

AmStem International is the name of the new company intended to result from the merger of Stem Cell Therapy International, Inc. and HistoStem, Ltd. of Korea. Histostem reportedly operates the largest accredited Cord Blood & Stem Cell bank in the world (more than twice as large as its nearest competitor) and is said to be already successfully treating patients with its patented, Korean FDA-approved stem cell therapies. Histostem is one of only a few stem cell companies in the world currently earning several million dollars in income from its cellular products and technology.

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CLINICAL

While desperately looking for enough cash to last beyond mon
th's end, Isolagen, Inc. (Amex: ILE) has managed to submit its Biologics License Application (BLA) for Isolagen Therapy, a cellular therapy for the treatment of wrinkles/nasolabial folds, to the U.S. Food and Drug Administration (FDA). The Company's wrinkles/nasolabial folds Phase III trials were conducted under an FDA Special Protocol Assessment. The company also recently completed its Phase II/III trial for the treatment of acne scars with statistically significant efficacy results. Meanwhile the company is actively pursuing financing and/or strategic partnerships including the potential sale of its 57% ownership interest in Agera Laboratories, Inc.

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TissueGene, Inc. announced that its South Korean licensing partner Kolon Life Science, Inc. (KLS) has received regulatory allowance from the Korea Food and Drug Administration (KFDA) to initiate a Phase IIa clinical trial of TissueGene-C (TG-C) in patients with severe osteoarthritis of the knee. TG-C has been developed for the localized delivery of allogeneic human cells expressing TGF-beta1 in order to induce the regeneration of cartilage. TissueGene is currently completing Phase I clinical trials in the U.S. and continues to work closely with KLS in order to coordinate their regulatory efforts and efficiently conduct parallel clinical trials for TG-C in both the U.S. and Korea. In Asia, TissueGene has licensed intellectual property rights to KLS, a subsidiary of Kolon, for the clinical development and commercialization of TissueGene's lead product candidates TG-C and TG-B, developed for the regeneration of cartilage and bone, respectively.
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The Gamida Cell-Teva joint venture announced that orphan designation was granted by the European Commission for the investigational medicinal product StemEx® for the treatment of acute lymphoblastic leukemia and acute myeloid leukemia. The European Commission consequently follows the positive opinion of the European Medicine Agency's Orphan Medicinal Products Committee. The FDA granted StemEx orphan drug designation in March 2005 for use as hematopoietic support in patients with relapsed or refractory hematologic malignancies (cancer of the blood system) who are receiving high dose therapy.

StemEx is a graft of expanded stem/progenitor cells, derived from a single unit of umbilical cord blood and transplanted in combination with non expanded cells from the same unit. It is currently being researched as an alternative to a bone marrow transplant, for patients with certain blood cancers, in an advanced Phase III, pivotal study called ExCell

By EU regulation, orphan medicinal products are intended for the diagnosis, prevention or treatment of life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or are medicines which, for economic reasons, would be unlikely to be developed without incentives. Benefits include, among others, market exclusivity in the European Union for 10 years from the date of the orphan medicinal product designation and an EMEA fee-reduction for the drug.

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Stemedica Cell Technologies, Inc., ("Stemedica") announced that they had received the results from a Stemedica-sponsored clinical study conducted by "renowned stem cell researcher and clinician" Professor Philippe Hernigou, MD, Ph.D. The "approved" clinical study is said to have involved treatment of non-union bone injuries (specifically tibia fractures) using autologous stem cells, allogeneic stem cells and comparative traditional treatment therapies. The results presented to the company by Dr. Hernigou were said (by Company leadership) to be "exciting and profound ... in comparison to other traditional treatments". The year-long study is said to have included sixty patients and part of an on-going, ten year study involving nearly four hundred patients by Dr. Herningou using stem cell therapy and traditional treatment of non-union bones and joints. Stemedica has conculded the study gives "clear evidence that the use of adult stem cells provided greater healing capability in a shorter period of time with less infection". What is not clear whether anyone else will be granted the privilege of looking at the data. This begs a number of questions. Do they intend to publish? What was the study design? What kind of study was it? What is Stemedica's intent for this data?

Having previously ran into some trouble with a scandal associated with the Premier of Bermuda, the company is busily attempting to reinvent itself by distinguishing itself from those other international stem cell clinics claiming to treat a host of diseases for a fee without registered clinical trials, publications, etc.

Here's what we know. In addition to their US operation they have "facilities" in Moscow, Russia and Seoul, Korea. They also have partnership with a hospital in Tijuana, Mexico providing stem cell treatments for Alzheimer's, Parkinson's, Multiple Sclerosis, Muscular Dystrophy, Stroke, Ischemic Brain Trauma, Spinal Cord Injury and other neurodegenerative conditions and diseases. They say they only provide their stem cells to "hospitals and research centers that are conducting studies under protocols approved by the appropriate regulatory agencies". They say their San Diego facility is "designed and constructed to be in compliance with appropriate FDA regulations including cGMP’s – Current Good Manufacturing Practice and GTP’s – Human Cells, Tissues and Cellular and Tissue-based products" and had CIRM leadership tripping over themselves recently after a visit their San Diego site to give the company and its facility glowing testimony (as reported in last week's blog). They say that they intend to conduct "future clinical studies in the United States".

Announcements like their recent proclamation of "exciting and profound" study results without publication are unlikely to dispel the critics that say this is more smoke masquerading as fire.

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Ok, this isn't "clinical" and I usually don't report on pre-clinical results but this one excites me and...it's my blog so skip to the next story if you don't wanna read it! (smile)

Opexa Therapeutics, Inc. (NASDAQ: OPXA) announced it has new preclinical data showing that cells obtained from peripheral blood of healthy and diabetic patients have the ability to differentiate - employing Opexa’s proprietary technology - into insulin-producing islet-like clusters demonstrating many of the expected characteristics of true pancreatic islet cells including the ability to secrete insulin, glucagon and somatostatin. Additional studies recently completed by Opexa’s scientists also reportedly support these findings showing high levels of C-peptide, a by-product of insulin synthesis, within these islet-like cells, as well as many key stem cell and pancreatic biomarkers. They also claim that in vivo studies involving the subcapsular kidney transplantation of these islet-like clusters into diabetic NOD/SCID mice resulted in a reduction in the blood glucose levels for prolonged periods of time.

Additional preclinical studies are planned to examine optimal dosing, delivery and route of administration of the islet-like clusters, and toxicology. Following a preliminary meeting with the FDA and through consultations with its Clinical Advisory Board, Opexa has designed a Phase I clinical study protocol. Primary endpoints are proposed to be safety of monocyte derived pancreatic-like islet cell transplantation and insulin independence or reduction in exogenous insulin requirements at one year post initial infusion.


COMMERCIAL

DNAmicroarray, Inc. in San Diego provides proprietary systems for controlling the differentiation of human stem cells. This is the low-hanging research tools business—the so-called shovels and blue jeans—to the scientists panning for gold. Still, while this is good business, DNAmicroarray president Babak Esmaeli-Azad told Xconomy San Diego editor, Bruce V. Bigelow, that he has also personally invested $2 million on internal stem cell research for potential therapies.

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Cryo-Save Group, the Netherlands-based stem cell company with a large Indian presence, plans to increase the number of stem cell donors in the country by opening more representative offices. Cryo-Save Group plans to invest €2 million (about Rs 13 crore) in its Bangalore-based subsidiary for over three years. Currently, the company is operating in Delhi, Mumbai, Pune, Ahmedabad and Bangalore. By the end of the year, they plan to open three additional centres, of which one will be in Kolkata, with a plan to expand into other South Asian countries by end of 2010. Cryo-Save currently charges Rs 75,000 per person to save a child’s umbilical cord blood and stem cells. This includes collection, processing and storing the cells for about 21 years.

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Just days after its purchase of Genentech, Roche has made another acquisition, albeit a much smaller one, of the German firm Innovatis. Roche has signed a definite agreement to buy the company for EUR 15 million. Innovatis provides automated cell analysis solutions, focusing particularly on cell counting, viability testing and cell function analysis in research and bioproduction. The deal is expected to be completed within the next few weeks.
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Lonza Group Ltd has announced that Anja Fiedler will replace Shawn Cavanagh as Head of Lonza Bioscience headquartered in Walkersville, MD. Anja Fiedler will become the new division head and member of the Lonza Management Committee (MC) as of 15 April 2009. Shawn, it is reported, "will leave the organization to pursue other interests". It does make one curious whether these other interests were what motivated him to leave or whether once he was shown the door we can only imagine he will now be motivated to find other interests. Whatever the case they did thank him for his dedication and wished him well in his future endeavors. Anja, curiously enough, comes to Lonza from outside the industry, most recently having been with Philip Morris. Is this a sign of more shake-ups to come as Lonza really starts to come to grips with its Bioscience acquisition now that it is fully integrated or is this just a one-off personnel changeup? Time will tell.

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The research tools sector - Life Technologies Corporation (NASDAQ:LIFE) and Sigma-Aldrich (NASDAQ:SIAL) in particular - are expected to report above estimated earnings given expected fallout of spending to occur from the Obama administration's recent decisions.

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Likely negotiating like its life dependended on it (oh ya, it does!),
Isolagen, Inc. (Amex: ILE) has announced that the United States District Court for the Eastern District of Pennsylvania has issued an order granting preliminary approval of an agreement to settle its securities class action lawsuit and its two derivative actions against the Company and certain of its current and former officers and directors.
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BioLife Solutions, Inc. (OTC Bulletin Board: BLFS) announced that an independent European comparison of the Company's CryoStor pre-formulated serum-free and protein-free biopreservation media against traditional in-house formulated culture media/serum/DMSO showed CryoStor offers a significant cryopreservation process improvement and better cellular outcomes. Compared to media/serum/DMSO, CryoStor enabled enhanced post-thaw cell membrane integrity and a full recovery of metabolic activity and differentiation capacity within 24 hours after thawing. The study findings also confirm that despite improved cell recovery immediately after thawing for media/serum/DMSO cryopreserved cells beneath alginate, up to 50 percent cell death still occurred within 24 hours post-thawing.

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Early-stage venture capital firm OVP Venture Partners (OVP) announced today that distinguished scientist Irving L. Weissman, M.D., will join its technical advisory group. Is this a signal of their intent to invest in stem cell plays you think?

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RESOURCES & EVENTS

What am I your secretary? Find your own resources and events! Kidding... sorry I'm a little punchy today. I simply didn't come across anything to include here that I thought was sexy enough for this sexy edition of Cell Therapy Blog.

Sign-off

Ever wonder why Cell Therapy News now only carries advertising from Stem Cell Technologies? Did it ever seem curious to you that they never include news from competitors of Stem Cell Tech's?

Could it be that Cell Therapy Blog is your only source for independent and objective news and analysis in the cell therapy industry?

Help spread the word ...

Friday, March 6, 2009

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 Charit√©-Universit√§tsmedizin, 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 Charit√© 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.