Cell Therapy Industry HiLites 2009-02-06

Following on the heels of FDA's approval for Geron's trial, I suppose it's not surprising that TIME magazine put stem cell research as its cover article. But while stem cell research enjoys the glory and any stock even remotely related to cell therapy is enjoying a bullish run, CIRM - one of the efforts responsible for keeping stem cell research alive in the US during the last 8 years - is being threatened by California's crushing debt.

It was an interesting week at the Phacilitate Cell & Gene Therapy Forum last week. In some ways it was a little Dickensian - the best of times; the worst of times. On one hand we had Genzyme-Osiris talking about the biggest deal ever in cell therapy, both Osiris and Dendreon potentially being on the verge of the first cell therapy BLA approval in a decade, and the pharma industry coming out of the closet speaking about their belief in cells as therapies not just tools for discovery or toxicity testing. On the other hand, the fact that money has pulled a disappearing act on many who thought they had it or been freeze-dried in the pockets of those who still do, makes it nervous times for a bulk of the industry. This is a sector that may look drastically different at the end of the year than it did at the beginning and while this will make for tough decisions, may impeded some progress, and generate many a sleepless night... an industry re-org may not be an entirely bad thing.
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This issue is sponsored by folks that sell stuff their customers honestly want to keep a secret.
If you knew what they know, you would know you can change the way you store your cells. You wouldn't be thinking the yield you're getting from your cellular source material and cryopreserved products is just fine - not worth investigating. You would know that BioLife's solutions (now both the subject of an FDA Master File) could very well improve your product, decreased product failures, and maybe even lower your costs. But then if you knew that, you'd already be a customer!

You probably owe it to your cells to find out what they know.

www.biolifesolutions.com

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I want to take a moment to correct something. In December I said that while I was a little confused by their recent press release, it looked to my like Wuxi-Apptec was getting out of the cell therapy manufacturing business. I took the opportunity at last week's Phacilitate meeting to get some clarification. Indeed, I was wrong. I asked WuXi AppTec, VP, Philadelphia Operations, Garry B. Takle, Ph.D. for clarification. I'm happy to share with you his reponse below:

The recent closure of the recombinant protein and monoclonal antibody manufacturing bioreactor suites in the WuxiAppTec, Philadelphia facility does not affect the continued focus on and expansion of cell therapy manufacturing, cell bank manufacturing and biologics testing programs at the site. WuXiAppTec has taken the strategic position that cell therapy products are likely to provide significant outsourcing revenue in the short and mid term, as these more novel products become firmly established and better accepted.

WuXiAppTec has been in the cell therapy business for more than 3 years and intends to use the experience gained during this period to attract a broader portfolio of cell therapy clients. WuXiAppTec has so far completed 5 cell therapy manufacturing projects, including both allogeneic and autologous cell therapies.

In addition, WuXiAppTec currently performs testing for more than 15 cell therapy clients and manufactures more than 50 cell banks per year. Combined with considerable experience in medical device testing and manufacture, and in the production of tissue-based products at the WuXiAppTec St Paul facility, the WuXiAppTec Philadelphia site provides the GMP/GTP cell expansion expertise to develop and manufacture a wide range of cellular therapies and combination products.

FINANCIAL

Cell therapy company Garnet Biotherapeutics, developing products for scar reduction after surgery or dermatological procedures, announced the close of a $10.4 million round led by SCP Vitalife Partners II, L.P. Garnet will use the proceeds to fund Phase II clinical trials of its proprietary human adult bone marrow-derived cells, as well as manufacturing and development. Safeguard Scientifics, Inc. and Alliance Technology Ventures also participated in the financing.

It is clearly too early to do the 3-count for Bioheart, Inc. (Nasdaq:BHRT) which announced private funding commitments of up to $7 Million. The company has entered into a non-binding letter of intent with Organic Business Alliances, LLC ("Organic") pursuant to which it would invest approximately $1.5 million in Bioheart under its current offering under Regulation D and, subject to Bioheart achieving certain specified milestones, invest up to an additional $5.5 million on terms substantially the same as those of the offering. The investment by Organic is subject to approval of the boards of directors of both Bioheart and Organic and mutually acceptable definitive agreements.

Pluristem Therapeutics Inc. (NasdaqCM:PSTI) (DAX:PJT), a bio-therapeutics company dedicated to the commercialization of non-personalized (allogeneic) cell therapy products for a variety of degenerative, ischemic and autoimmune indications, announced that on January 29, 2009, the Company entered into definitive agreements to sell restricted common stock and warrants for aggregate gross proceeds of $1,200,000.

The company also recently announced that it had received approval (in the form of a declaration) from Biotec Services International Limited, stating that Pluristem's Investigational Medicinal Product (IMP), PLX-PAD, is manufactured in accordance with standards of Good Manufacturing Practice (GMP), equivalent to those applied in the EU. Biotec Services is an international clinical trials supply company providing release services of the IMP into the European Union by a Qualified Person (QP). Upon IND and IMPD clearance by the regulatory authorities, Pluristem plans to initiate clinical trials in Europe and in the U.S.A., administering PLX-PAD to patients afflicted with critical limb ischemia, which has not responded to traditional medical or surgical interventions. They've invested ~$2M building in their facility and team to get them to the point of a successful audit of the manufacturing site by a Qualified Person. <more>

Following the implementation of the Clinical Trials Directive 2001/20/ EC in May 2004, any company wishing to undertake European clinical studies must have a Qualified Person (QP) release supplies of Investigational Medicinal Product (IMP) that have been manufactured outside the EU for use in European clinical studies. The QP conducts audits of manufacturing facilities outside the EU, ensuring that each batch has been manufactured in accordance with current Good Manufacturing Practices (cGMP) guidelines.

Organogenesis, Inc has been awareded a two-year, $7.4 million grant from the Massachusetts Life Sciences Center to support the firm’s expansion in the state. Organogenesis, a Canton, MA-based maker of living cell therapies for damaged tissues, has purchased two buildings in Canton and plans to grow its Massachusetts workforce from 220 today to 500 workers, according to the Life Sciences Center. Apligraf® has been used to treat many thousands of patients and is covered extensively by Medicare, Medicaid and by 350 private payers in the U.S.

According to their recently filed Form 10-Q (Quarterly Report), Aastrom Biosciences (Nasdaq:ASTM) executed a $15 million common stock purchase program with Fusion Capital Fund II, LLC (Fusion Capital) on October 27, 2008. They have the right, over a 25-month period, to sell shares of common stock to Fusion Capital from time to time in amounts between $60,000 and $2 million, up to an aggregate of $15 million, when they choose to do so, based on the terms of the agreement.

In an accompanying press release, Aastrom outlined a number of recent and anticipated events including the fact the FDA put their dilated cardiomyopathy (DCM) trial on hold Feb 2 for reasons which appear associated with the anesthesia administration.

ThermoGenesis Corp. (NASDAQ: KOOL) has reported second quarter results in whixh the company records the second quarterly sequential increase of 36%. For the quarter ended December 31, 2008, the Company reported revenues of $6.1 million, a 12 percent increase over revenues of $5.5 million in the same period a year ago. Disposable revenues in the quarter were $3.5 million, a 35 percent increase over disposable revenues of $2.6 million in the second quarter of fiscal 2008. Disposable revenues associated with the Company’s core AXP AutoXpress Platform™ (AXP™) and BioArchive® Systems offering increased by 56 percent year-over-year as AXP bag set volume in the quarter was 26,500 versus 20,000 in the second quarter a year ago.

The ThermoGenesis' MarrowXpress (MXP) is now being used to concentrate stem cells from bone marrow in an ongoing Phase II clinical trial being conducted at the University of Naples in Italy to study the effect of bone marrrow derived mononuclear cells on patients with critical limb ischemia (CLI). <more> <analysis>

Millipore Corporation (NYSE: MIL), a provider of technologies, tools and services for the global life science industry, announced that it has entered into a $22M agreement to acquire Guava Technologies, a provider of bench top cell analysis systems. The acquisition follows the distribution and co-development partnership the two companies announced in March 2008. Millipore says that its recently announced deal to acquire Guava Technologies will let it develop new kits for stem cell research. <more>

BioLife Solutions, Inc. (OTC Bulletin Board: BLFS), a leading developer and marketer of proprietary GMP hypothermic storage and cryopreservation media products for cells, tissues, and organs, announced record revenue and new funding to support internal manufacturing scale-up already underway to support increased product demand. The company registered record revenue for the fourth quarter and full year 2008. Preliminary fourth quarter 2008 revenue was $417,818, an increase of 36 percent from the third quarter of 2008, and 40 percent over the fourth quarter of 2007. This includes initial orders from nearly 30 new customers. Preliminary full year 2008 revenue was $1,326,772, an increase of 36 percent over 2007 fiscal year revenue.

Biolife also landed $1.4 million in credit funding from two shareleholders for operations and the completion of construction and validation of a new manufacturing facility.

To complete its triage of announcments, the company announced the launch of its new quarterly newsletter BioPreservation Today. They hope the publication will be a forum for sharing knowledge about optimizing biopreservation protocols and processes, to maximize the yield and function of cells and tissues in research and clinical applications.

Healthcare of Today, Inc ("Healthcare of Today"), announced the $100M acquisition of Regenetech, Inc ("Regenetech"). The Regenetech acquisition is one of a series of major acquisitions made by the holding company recently. Last week Healthcare of Today announced that it had obtained Advanced Developers, a builder of resort-like senior facilities in production in Florida and South Carolina. Prior to that, the organization had entered into an agreement to acquire another $19.4M medical facility in Agoura Hills, California.

Healthcare of Today, formed in May of 2008, is a holding company focused on acquiring and developing companies primarily within the healthcare industry. Through its subsidiaries, Healthcare of Today owns a variety of businesses including: nurse staffing, insurance, pharmaceuticals, residential care facilities for the elderly, assisted living facilities, skilled nursing facilities, senior communities, home healthcare services, senior fitness, real estate brokerage/relocation services, adult daycare, a private chef network, a culinary institute, healthcare information technology, nurse education, and biotechnology.

Regenetech® has concentrated its research efforts on development of umbilical cord blood and peripheral blood expansion, increased regeneration of bone for orthopedic uses, and production of biomolecules. Regenetech®’s business purpose is to commercialize adult stem cell expansion technology by developing stem cell and tissue regeneration technology, patenting it quickly and either licensing or establishing joint ventures to commercialize the technology to generate revenue.

Last year Regenetech announced it could achieve a 60-fold increase of adult stem cells through its cellXpansion™ process, namely CD34+38- peripheral blood progenitor cells (adult stem cells). These results were reportedly achieved through the continued development of cell culture conditions in Regenetech’s proprietary and patented Intrifuge™ cellXpansion™ technology. As I recall from listening to a presentation last May, the core technology is essentialy an electro-magnetic field placed around a bioreactor.

The company was founded in April of 2002 as BioCell Innovations. In May of 2002 the company acquired its original patent licensing rights through a novation from NASA. In May of 2002 Regenetech® entered into a Sponsored Research Agreement with M.D. Anderson Cancer Center in Houston, Texas. Regenetech® filed its first patents in August 2002. The company name was changed to Regenetech®, Inc. in November of 2002 in order to begin developing trade name value. Stage 1 research was fully completed in January 2003. Regenetech® licensed its first intellectual property for treatment of Sickle Cell Anemia. In April 2004 Regenetech® Licensed an additional two (2) patents from NASA relating to advanced bioreactor technology.
In April 2004 Regenetech® entered into a Reimbursable Space Act Agreement with NASA to continue development of adult stem cell expansion. In January 2005 Regenetech® confirmed its significantly expanded adult stem cells were free from cancer risks and had no genetic modification. In 2005-2006, Regenetech® expanded its research at NASA and several universities and completed the filing of its first73 patent applications.

The company's former CEO Dr. David Bonner, who appeared at one point to be steering the business toward involvement with off-shore commercial stem cell transplant clinics, had a parting of ways with the company a year or so. He then opened up Stematix, Inc with some of his former team. Stematix is in business to "support and establish fully operational stem cell banks and therapy clinics around the world."

Intercytex Group plc (LSE: ICX) ("Intercytex" or "the Company"), a developer of regenerative medicine products, has implemented restructuring actions to reduce cash burn. Despite the very positive progress being made on all projects, with the financial markets in their current state, the Company will shortly commence a 30 day consultation period with employee representatives proposing a reduction in headcount at the Company's facilities in Manchester UK and Boston, USA of around 50% from the current level of 76 employees. The reduction in workforce will extend the Company's existing cash resources to fund operations until around the end of 2009. The measures are expected to be implemented with effect from March 2009.

CLINICAL

Advanced Cell Technology, Inc. (OTC:ACTC) provided a corporate update in which it announced that it has accelerated its efforts to file an IND for its retinal pigment epithelium (RPE) cells program for the treatment of diseases of the eye in light of today’s decision by the U.S. Food and Drug Administration to grant clearance for a stem cell company to begin trials for the world's first study of a human embryonic stem cell-based therapy for people. The Company is close to securing the necessary funding which would allow it to make the requisite filings during the summer of this year

Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced today that its collaborators at the University of Pennsylvania (Carl June) have opened a Phase 1 clinical trial to evaluate SB-728-T for the treatment of HIV/AIDS. SB-728-T is produced by specifically modify the patient's own CD4+ T-cells, the principal target of HIV infection, with a one-time exposure of the cells to CCR5-specific ZFNs. This generates a population of T-cells that lack the CCR5 receptor, are resistant to HIV and can be infused back into the patient to provide a reservoir of HIV-resistant functional immune cells and, more importantly, may expand and provide an HIV immune response. specifically modify the patient's own CD4+ T-cells, the principal target of HIV infection, with a one-time exposure of the cells to CCR5-specific ZFNs. This generates a population of T-cells that lack the CCR5 receptor, are resistant to HIV and can be infused back into the patient to provide a reservoir of HIV-resistant functional immune cells and, more importantly, may expand and provide an HIV immune response." The primary objective of the study is to evaluate the safety and tolerability of SB-728-T. In addition to safety monitoring, data will be collected on the expansion and persistence of ZFN-modified cells, CD4+ cell counts and viral load.

According to a online publication in Molecular Therapy, Stratatech Corporation has engineered a skin substitute with potent anti-infective function. The product is a genetically modified version of its StrataGraft® living human skin substitute designed to actively fight costly bacterial infections that routinely develop at the site of burns and other severe skin injuries, such as skin ulcers. Stratatech’s skin-substitute innovation reportedly enables the immediate topical delivery of a potent anti-infective that actively inhibits bacterial growth and promotes regrowth of the patient’s own cells. The anti-infective capacity of Stratatech’s genetically-engineered tissue, which is being developed and commercialized by the company as ExpressGraft™ Enhance skin substitute was generated using a non-viral vector, or carrier. The company believes it is the first time a virus-free approach has been used to genetically modify a living, cell-based tissue substitute

Therakos, Inc. announced that Health Canada has cleared the THERAKOS(TM) CELLEX(TM) 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.

Stem Cell Innovations, Inc. (OTC Pink Sheets: SCLL) appears to be working on a liver assist device to support patients waiting for transplant. The company has not made a formal announcement that I can find and the company's website is woefully out-of-date but the device was described in a paper published in the most recent issue of Current Gastroenterology Reports. Medical News Today was able to get the company to comment for an article they did highlighting the article: "There are currently three times as many patients on the waiting list for liver transplantation as there are organs available and few ways to stabilize them if they enter an acute phase of their disease," said Kelly, CEO of Stem Cell Innovations. This second generation device, based on the PluriCells, "combines the safety of primary hepatocytes (normal liver cells) with the standardization of a cell line," continued Kelly. "We're building on what we learned from our earlier device which utilizes the C3A cells that form the basis of Stem Cell Innovations' ACTIVTox(R) drug discovery products. We are one of the few companies that can approach this problem from both sides: from the stem cell and from the adult liver cell. We are using our PluriCell(TM) system to understand the requirements of the liver stem cell and our ACTIVTox system to understand the requirements of the mature cell."

COMMERCIAL

Privately held SkinMedica of Carlsbad says it has filed a patent infringement lawsuit against new San Diego startup Histogen and Gail Naughton, its founder and CEO. The lawsuit, which was filed in federal court in San Diego, alleges that Histogen and its affiliated Histogen Aesthetics business infringe on two SkinMedica patents related to proprietary conditioned cell media technology. SkinMedica says its complaint asks for unspecified relief for the “misapprorpiration of SkinMedica trade secrets and confidential information by Histogen.”

International Stem Cell Corporation (OTCBB: ISCO) continued its recent publicity campaign with a letter to shareholders outlining recent development and plans for the company and their human parthenogenetic stem cells. The company is thrilled to have had their name dropped in a recent article in the Economist along with corporate pioneers like Geron & ACT.

Neuralstem Inc. (AMEX: CUR) has nailed down a key patent for its core technology, which grows neural stem cells from the brain to replace damaged spinal cord cells. The patent comes in time for Neuralstem’s first clinical trials, planned for this year if the Food and Drug Administration gives them the green light. The Rockville company intends to test the ability and safety of injecting fetal stem cells into the spinal cords of patients suffering from amyotrophic lateral sclerosis, more commonly known as Lou Gehrig’s Disease. Neuralstem filed its request in December to launch its first human clinical trials this spring. It expects to hear the FDA’s decision by the middle of next month, but another competitor’s recent news has raised its hopes.

Bio-Matrix Scientific Group (OTCBB: BMSN) (www.BMSN.us) has entered into a cell processing and storage contract with both NeoCells, Inc. and AdultCells, Inc. (ACI; www.adultcells.com). Pursuant to this Contract, BMSN has agreed to (a) cryogenic banking and processing of umbilical cord blood and adult peripheral blood stem cells of clients from both NCI and ACI and (b) cryogenic banking and processing of menstrual blood and other human stem cells of clients from both NCI and ACI upon BMSN’s approval. Fred Fitzsimmons, CEO of NeoCells, Inc. and AdultCells, Inc., envisions “the developing relationship between BMSN, NCI and ACI leading to the establishment of the premier stem cell cryogenic storage company in the U.S.” he said.

RESOURCES & EVENTS

Ex-FDA regulatory consultant Darin Weber (aka "Regenerative Medicine Guru") now of the Biologics Consulting Group, has posted on his blog a number of useful presentations and articles he did in 2008. It's worth checking out.

You won't find a press release about this anywhere but the only weekly publication dedicated to news in the cell therapy sector, Cell Therapy News (which I founded back in 2001 as an email to colleagues and grew to ~11,000 subscribers) has now been subsumed by the Stem Cell Technologies' marketing department. The publication is no longer accepting outside advertising. Cell Therapy News is a news amalgamation service and does not do original and/or editorial content. The publication's objectivity should not be compromised unless they refuse to include press releases from competitors. Rumor has it this is indeed the case. Disappointing if true.

If you're interested in getting into the mind of Stem Cell Technologies' owner and founder, Allen C. Eaves, you'll find the rambling interview of him recently posted by Q1 to be an interesting read.

Monya Baker from The Niche, always does such a nice job of collecting primary and divergent sources on a topic. She's done the same on her second post about Geron. If this is a trial, company, or stock in which you are particularly interested, you'll want to check out her collection of articles.

This past week's GEN podcast was "Stem Cell Therapies on the Rise" by Linda Powers, Cofounder and Managing Director of Toucan Capital, which has 16 stem cell or regenerative medicine companies in its portfolio. Linda specifically addresses the issue of how the FDA's decision on Geron's IND might boost the development of stem cell therapies and speculates on what may have influenced the agency's decision to give Geron the go-ahead for a trial at this time. Powers also discusses why all the attention on human embryonic stem cell research has given short shrift to work with human adult stem cells and provides specific examples of the successful application of adult stem cells to treat human diseases. Powers describes what's needed from the financial, political, regulatory, and public acceptance arenas to make stem cell and regenerative medicine part of an expanded armentarium of therapeutic approaches in applied and clinical medicine. She also explains why the establishment of a biomanufacturing industry for the production and processing of stem cells and other living cells could give rise to a new and significant business segment of the American economy.

NIH/NHLBI and PACT have announced a workshop on 23-24 April entitled "Converging Concepts in Cell Therapy". This workshop will be held at the National Institutes of Health (NIH), Natcher Conference Center in Bethesda, Maryland.

University of Minnesota is hosting a professional short course, "Preservation of cells, tissues and gametes" to be held May 18-20, 2009.

Sign-off...

I was very pleased to see a 1.5-page article entitled "Stem cell alliance" in the January 2009 issue of R&D Directions magazine, a very pharma-focused publication related to 'pharmaceutical research and development'. The >$1B deal Genzyme-Osiris deal has caught the attention of many in the pharma sector who heretofore have believed the sector too immature to mean real business.

That ends a hectic two-weeks. Sometimes conferences and paying work get in the way of blogging. I can't really complain. But that's why this is later than my usual Friday pub date. I have every intent to get back to the weekly schedule on Friday. We've had a good run of new subscribers sign up this week. Welcome ya'll. If you learn anything here we hope it's that cell therapy...means business! :)