As Late, Late Show host, Craig Ferguson, says..."It's a great day for America. 'Why is it a great day for America,' you ask? Well, let me tell you".
The US House of Representatives has passed what promises to be (if approved by the Senate) the largest single budget increase for the FDA in the agency's history. Now many an economist or financial wizard may argue why this is fiscally irresponsible given America's economic woes but it certainly is promising for the biotech industry which has suffered due to a lack of resources in the Agency overseeing the sector. This lack of resources permeates into almost every interaction with the agency whether it be awaiting new Guidance, submission reviews, staff attrition, etc.
As it happens it is also a great day for the mother country from which America declared its independence because of their dastardly tea-taxing ways. "Oh, why ever it that so?", you might ask with a fake English accent. Well I'lltell you. It's because the UK Office for Life Sciences (OLS) has announced a "Life Sciences Blueprint" that will attempt to capitalise (well, that's how the English spell it) fully on its "leading global position in regenerative medicine". The OLS has announced £18 million funding from the Technology Strategy Board for a Regenerative Medicine programme (again with the English spelling) of investment to support key areas of commercial R&D and the development of R&D partnerships, supported by additional funding of £3.5 million from the Medical Research Council, the Engineering and Physical Sciences Research Council and the Biotechnology and Biological Sciences Research Council.
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I fell in love with this conference, the venue, and the charming city of Leipzig when it was held in 2007. For a unique and international take on the multi-disciplinary world of regenerative medicine, you should consider meeting at the WCRM 29-31 October 2009.
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Well I seem to be on a roll with anything related to Wake Forest recently so 'why stop now' I ask myself, without any compelling reply to the negative. This is just a little FYI. The Babcock Demon Incubator (BDI), a new venture incubator at WFU, promises to be a unique new venture incubator opportunity for regenerative medicine startups. The BDI has has fully equipped wet lab space with available equipment including chemical and tissue culture hoods, PCR, Gel systems, HPLC, microscopes, centrifuges, etc. In addition, BDI clients have access to collaborative opportunities at the Wake Forest Institute of Regenerative Medicine and to a wide range of services and specialized equipment. The incubator provides both residential and non-residential client models depending on client needs. All clients have access to office or lab space, can take advantage of business consulting, and access to an extensive services network designed to provide difficult-to-obtain resources to help young companies succeed. For more information, visit the BDI web site.
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As regular readers of my Industry HiLites will know, from time-to-time I occasionally profile a company here in the front-end of the post providing some background, analysis, and recent developments. I'll formalize this a little by calling it a COMPANY PROFILE but won't promise to include one every time.
On another house-keeping matter you will note that I've changed the section title "FINANCIAL" to "FINANCIAL and DEALS".
COMPANY PROFILE
Bioheart, Inc., (OTC Bulletin Board: BHRT) has announced that they will proceed to the final phase of their clinical trial with muscle stem cells for treatment of cardiovascular disease.
The company convened a panel of experts to review pre-clinical data dating back to 1988 and clinical data from controlled studies which began in early 2000 (involving nearly 400 patients). Bioheart’s latest Phase II/III MARVEL study has undergone interim analysis. While the data has not been disclosed pending publication confidentiality, this panel of experts was reportedly asked to also review this data.
The panel is reported to have "unanimously and enthusiastically endorsed moving forward" to complete the final phase of clinical trials (MARVEL) to advance muscle stem cell (myoblast) therapy for treating heart failure with the goal of bringing the product to market with FDA approval and CMS reimbursement.
It's not entirely clear to me how or why this panel of experts was called together but a listen of the audio of the panel discussion frankly makes it sound a little bit like a love-in for Bioheart. In the accompanying slide deck, you will find an overview by Chairman and CEO, Howard J. Leonhardt listing an extremely aggressive list of deliverables for 2009 for a dizzying array of clinical products & programs.
On the financial front, Leonhardt states the company has to-date invested $125 million to support heart failure-related research - $85 million of which was in paid capital.
From its May 20, 2009 Form 10-Q Quarterly Report filed with the SEC, "The Company has incurred significant operating losses over the past several years and has a deficit accumulated during the development stage of $98.6 million as of March 31, 2009. In addition, as of March 31, 2009, the Company’s current liabilities exceed current assets by $12.7 million. Current liabilities include notes payable of $5.4 million. ...the Company also has an obligation of $3 million to the Company’s Chairman and spouse [they repaid the principal on a company loan and a pro rata portion of accrued interest ]. The Company does not have sufficient cash to support its operations through December 2009. The Company will need to secure significant additional sources of capital in June 2009 to develop its business and product candidates as planned."
On July 9, the company issued a press release saying that between October 1, 2008, and July 7, 2009, Bioheart received proceeds in the amount of $2,855,830 from the placement of restricted common stock and warrants under its current offering which it has now extended through October 2009. There's a little positive spinning here because this would appear to include the approximately $1.8 million raised in the sale of stocks and warrants which it announced back in October 2008. The new ~$1M appears to be coming in dribs and drabs from the sale of small chunks of stock or warrants including, if I'm reading it right, the granting of stock in repayment of an outstanding loan (see the May 2009 Quarterly Report for clarification and details).
FINANCIAL and DEALS
Bioheart, Inc., (OTCBB: BHRT) has raised approximately $1 million over the past few month in an ongoing offering (see details above).
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Continuing on its very active deal-making course to becoming one of the premier sources of stem cell lines, media, antibodies, cultureware, and characterization kits in the pre-clinical end of the cell therapy sector, Millipore Corporation has signed a co-marketing deal with BioTime, Inc. (OTCBB:BTIM) subsidiary Embryome Sciences, Inc whereby Millipore will become a worldwide distributor of the ACTCellerate™ human progenitor cell lines derived from human embryonic stem cells but not fully differentiated into specific cell types
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Not to be outdone by its sub, BioTime, Inc. (OTCBB:BTIM) announced that two of its shareholders have completed the funding of the second tranche of their equity investments in BioTime following the exercise of their rights to purchase additional shares and warrants under the terms of their May 2009 stock and warrant purchase agreements. Broadwood Partners, L.P. and George Karfunkel have each purchased an additional 1,100,000 BioTime common shares and 1,100,000 stock purchase warrants. BioTime received $4,000,000 from the sale of the additional shares and warrants. The warrants, which are substantially the same as BioTime’s publicly traded stock purchase warrants, entitle the investors to purchase additional common shares at an exercise price of $2.00 per share. The warrants will expire on October 31, 2010 and may not be exercised after that date. “The $8,000,000 of new equity capital we have raised since May from these investors, plus our $4,700,000 research grant from the California Institute for Regenerative Medicine, will be available to finance our strategic programs to build our product and technology portfolios in the emerging fields of stem cell research and regenerative medicine,” said Michael West, Ph.D., BioTime’s CEO. **
America Stem Cell, Inc. has been awarded $2.5 million from the Texas Emerging Technology Fund to use in developing its enzyme technology platforms (ASC-101 and ASC-201) designed to improve the homing and engraftment of stem cells to target organs and increase their therapeutic potential for cancer patients. Additionally, these platforms have the potential to enhance stem cell treatment of inflammation from chemotherapy/radiation, solid tumors, autoimmune diseases, and ischemic diseases including myocardial infarction and stroke.
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Aastrom Biosciences, Inc. (Nasdaq:ASTM) has gained a modest extension from NASDAQ to comply with the $1.00 minimum closing bid price rule in order to remain listed on the Nasdaq Capital Market. This means they now have until October 1, 2009 to regain compliance by achieving a $1.00 closing bid price for a minimum of 10 consecutive trading days anytime before that date. If not, they get kicked off the NASDAQ and forced to find another market for their listing. Absent a stock split it seems an unlikely goal give that the stock is currently trading in the region of .35 cents and the 52 range for the stock is 0.15 - 0.78.
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Quantum Immunologics Inc., a Tampa, Fla.-based developer of cancer immunotherpeutics, has raised $2.2 million in VC funding from Mentor Capital, Inc. (Pink Sheets:MNTR). In addition to providing $2.2 Million in funding to help support the FDA trials through approximately February 2010, Mentor Capital has agreed to assist QI in funding future strategic stock or cash acquisitions. Mentor will also stand by as a preferred funding source for QI during later stage or additional trials. This commitment, of course, stands to reason given that QI represents the majority of Mentor Capital’s portfolio assets.
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The good news for StemCyte, Inc recently bagged $1 million in funding; the not as good news for StemCyte, Inc. is that it is a debt funding round from debt financing firm Agility Capital. Agility said that the senior secured growth capital loan closed in June. The firm has previously received venture funding from Sycamore Venture Capital, Asia Star IT Fund and National Technology Enterprise Co. of Kuwait, and the W.I. Harper Group. Agility is a provider of venture debt to high technology and other companies. This blogger speculates the debt financing will be used to fund the company's commitments to a recently announced joint venture, StemCyte Therapeutics India Pvt. Ltd, which is expected to be revenue-generating in the near-term.
CLINICAL
Quantum Immunologics, Inc, a Tampa, Florida company focusing on the research and development of cancer immunotherapies, announced that the first patient in its breast cancer trial has begun to receive its dendritic cell therapy. QI is currently sponsoring and conducting an FDA-authorized Phase I/II clinical trial testing the safety and efficacy of its immunotherapy on 27 Stage IV breast cancer patients who have failed conventional therapy. Each patient will receive three monthly injections of the patient's own dendritic cells that have been sensitized to OFA (oncofetal antigen) - a cancer antigen (a protein found on cancer cells that can be targeted by the body's own immune system) found in many tumor cell lines or fetal tissue, but absent on normal, healthy tissue. When the sensitized cells are injected back into the patient, QI expects the patient's T-cells will locate the OFA found on the patient's cancer cells, thereby generating an immune response with the goal of killing the cancer cells and preventing further spread of the disease.
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Harvest Technologies Corp. announced that the company-sponsored 60-patient clinical trial conducted in Chennai, India using the company's BMAC point-of-care System to treat patients with non-reconstructable Critical Limb Ischemia (CLI) has completed enrollment. Over 300 CLI patients have been treated with the Harvest BMAC treatment protocol in an ongoing multi-center FDA study in the U.S. and other studies in India, Germany and the Czech Republic.
COMMERCIAL
Dendreon Inc (NASDAQ:DNDN) is currently eying Atlanta, GA as the location for a new $80 million, 300-person manufacturing facility. "Why Atlanta", you ask? "Ah, good question", I say. It all comes down to transportation logistics. An autologous cell therapy like Provenge requires the acquisition of a biological sample from the patient which is then used to manufacture a unit of the therapeutic that is then shipped back to the patient's clinical stie for administration. This makes logistics a primary consideration for the business model. Atlanta, has one of the world's busiest and most connected airports.
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Cord Blood America, Inc. (OTCBB:CBAI) has signed a lease for a 17,000 square foot building in Las Vegas, Nevada, for a state-of-the-art laboratory for the storage of multiple stem cell products including umbilical cord blood stem cells. Aggressively the company intents to start processing and storing its customer's umbilical cord blood stem cells in the facility in the fourth quarter of 2009. According the the company CEO, in 2010, CBAI intends to expand its cryogenic storage services to other forms of stem cells, including peripheral blood stem cell and adipose tissues. Presumably this move to self-manufacture will mean that its Corcell banking brand will no longer be able to claim the advantage of cGMP-quality processing and storage which it has made a part of its pitch since contracting with Progenitor Cell Therapy in 2007.
Meanwhile CBAI has picked the pockets of Bio-Matrix Scientific Group Inc (OTCBB:BMSN) by hiring its former Vice President and Chief Operating Officer Geoffrey John O’Neill as its new Laboratory Director and Brian Pockett as Vice President of Laboratory Operations.
In its May 2009 quarterly report, Bio-Matrix Scientific Group Inc (OTCBB:BMSN) reported they had all of $6,621 cash on hand with current liabilities of $1,371,834 such liabilities consisting of Accounts Payable, Notes Payable, Accrued Payroll Taxes, Amounts due to Shareholder, Accrued Expenses and Accrued Interest. Additional financing will be required to meet cash requirements over the next twelve months.
Meanwhile in a weird twist of almost Seinfeld-like interconnectedness, in researching SEC filings for this post I discovered that on August 11, 2008, MSN entered into a Letter of Intent with CBAI regarding the processing and storage by BMSN of cord blood specimens newly acquired by CBAI for consideration acceptable to BMSN. It would appear, however, that deal never materialized given CBAI's recent moves.
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Living Cell Technologies Limited (ASX:LCT; OTCQX:LVCLY) has announced the formation of a subsidiary, LCT Biomedical Limited in Russia, to facilitate the commercial development of DIABECELL®, its lead product for the treatment of type 1 diabetes. Reportedly the regulatory process has already been initiated and it is envisaged that the pivotal study and product would be registrable by late 2011. They recently implanted their 8th patients with DIABECELL, their porcine xenogeneic cell therapy product in clinical trial for type 1 diabetes. The plan is for the product to be manufactured and supplied from New Zealand, where the company is located, for the foreseeable future.
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Progenitor Cell Therapy, LLC has appointed a Vice President of Manufacturing Operations with extensive pharmaceutical production experience to head its two U.S. contract manufacturing facilities. PCT recent announced the recent appointment of Daryl LeSueur as Vice President of Manufacturing Operations. As head of Manufacturing Operations, Daryl is responsible for managing and supervising the day-to-day conduct of the manufacturing, packaging, and operational functions of PCT’s two North American contract manufacturing facilities.
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TheraCell, Inc. aims to develop a revolutionary point-of-care device for isolating and processing stem cells that will facilitate the broad use of autologous stem cells combined with an injectable, highly oxygenated scaffold gel for for advanced regenerative medicine procedures such as spinal fusion. The company recently licensed the underlying technology for the planned device from Yissum Research Development Company of the Hebrew University of Jerusalem Ltd., the technology transfer arm of the University. The technology was invented by a team headed by Professor Dan Gazit from the Faculty of Dental Medicine at the Hebrew University of Jerusalem.
Included in the license was a special scaffold with a particularly high oxygen-carrying capacity, which aims to increase fusion rates by increasing local oxygenation at the surgical site. Scaffolds derived from this technology will act to improve oxygenation and encourage the activity of stem cells and bone-forming cells, or osteoblasts. In preclinical experiments in rodents, accelerated spinal fusion and bone fracture healing were observed, as well as improved stem cell survival. The licensed technology also includes a novel and efficient methods for purifying and manipulating stem cells as well as an innovative, biologically compatible, oxygenated gel that improves bone regeneration and fusion of bone grafts
The company intends to pursue a medical devise status for the combined technologies which would be faster than if it were deems a biologic or combination product.
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In response to Cytori's Request for Designation, Cytori Therapeutics, Inc. (NASDAQ:CYTX) has been informed by the U.S. Food and Drug Administration (FDA) that its Celution(R) 700 System will be regulated as a medical device and reviewed by the FDA's Center for Biologics Evaluation and Research under the law applicable to medical devices. Cytori can now begin to compile and submit a marketing application to the FDA for the Celution(R) 700 System for use as a medical device in "aesthetic body contouring and/or filling of soft tissue voids" - in other words, "breasts". Before they get that far, however, there is still the unresolved issue of whether they will require further clinical trials. Clinical investigations, if required, would be conducted in accordance with the Investigational Device Exemption (IDE) regulations, not the Investigational New Drug regulations applicable to drugs and biologics
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New England Cord Blood Bank, Inc. (NECBB) announced that in August it will begin processing cord blood units using the fully closed and sterile AutoXpress™ System (AXP), developed by ThermoGenesis Corp. and distributed by GE Healthcare. They will, however, continue to operate their manual system, which will allow for processing of samples too small for the automated system. The company hopes that implementation of the automated system will bring NECBB one step closer to accreditation as a public cord blood bank in addition to offering its existing private banking services.
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Advanced Cell Technology, Inc. (Advanced Cell, ACT) (OTC: ACTC) announced that it has filed its Form 10-K for the year-ended December 31, 2008. The Company anticipates filing its Form 10-Q for its first quarter ended March 30, 2009 prior to the end of July. Upon completing that filing, the Company intends to file for relisting on the Over-the-Counter Bulletin Board as its financial statements will be current.
MISCELLANIA
The ERA Consulting Group is proud to announce the next intERActions seminars entitled:
The Regulation of “Advanced Therapy Medicinal Products*” in Europe, from the Clinic to Approval (*Cell Therapy, Gene Therapy and Tissue Engineered Products)
Topics to be covered:
* The EU regulatory framework for Advanced Therapy Medicinal Products (gene, cell therapy and tissue engineered products)
* Opportunities in the EU regulatory landscape, especially for rare diseases: How can they benefit your company?
* Current and future regulatory guidelines: Latest developments and implications for your company and product portfolio
* What are the EU CMC/quality issues/requirements at various stages of development?
* Preclinical development program: EU regulatory expectations - rational approach
* Clinical trials with Advanced Therapy Medicinal Products in the EU
Including Europe in your Strategy to Add Value to Early-Stage Biotech Product Development
Topics to be covered:
* The value of including an EU component into your overall regulatory strategy
* Optimizing interactions with European regulators and how this can aid development and add value
* The benefits of orphan medical product designation in the EU and an explanation of the procedure
* Clinical Development in the EU: The European Clinical Trials Directive
* Opportunities in the EU regulatory framework to add value and reduce time to approval, such as accelerated review, conditional approval or exceptional circumstances
* Compassionate use programs
The seminars will be held at The GreenV Sustainable Center of South San Francisco on 14th – 15th September 2009. For a complete program and registration information, please visit their website at: www.eraconsulting.com.
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After 15 years in the biotech conference industry, the Williamsburg BioProcessing Foundation has shuttered its operation in financial distress. Hasta la vista, Wilbio.
Sign-off
Ok, this is a stretch for inclusion in this blog which is why I leave it to this humble location in the post but two fellow Canadians have been named among the six researchers to receive prestigious awards from the American Society of Hematology later this year. Congratulations to stem cell researchers Connie Eaves and John Dick.
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Last week I mentioned the Regenerative Medicine Foundation and the conference they have announced for Spring 2010. I promised to find out more and bring you what I learned.
I've discovered that the driver behind the RMF is Anthony Atala and Case Western. It is a 501(c)(3) charity, non-profit organization. It was formed in 2005 by Dr. Atala to host a meeting with the U.S. Food and Drug Administration (FDA) around the topic of regenerative medicine. In 2006, the Foundation was instrumental in the formation of STRAC, the Soldier Treatment and Rengeneration Consortium, a national partnership of leading military and academic research centers and industry. STRAC formed the basis for the Armed Forces Institute of Regenerative Medicine (AFIRM).
Now the Foundation announced the first annual "Translational Issues in Regenerative Medicine" Conference, to be held in Winston-Salem, NC, December 8-10. The conference promises to focus on best practices in clinical trials and GMP facilities, as well providing a forum to discuss regulatory and reimbursement challenges. In addition, the conference will feature a venture forum to introduce early-stage and later-stage companies to accredited and institutional investors.
As its name suggests, the organization functions like a "foundation" rather than a member-based organization. This is reinforced by the fact the "Participate" page on the website has no content.
All of this is interesting but nothing more than you would learn from the RMF's website. What I DID discover this week that isn't obvious from anything yet online is that Atala has also been a primary driver behind the creation of a newly announced organization, this one a 501(c)(4) organization intended to be much more about lobbying.
This week the Alliance for Regenerative Medicine was formerly launched. (no website yet). Headquartered in Washington, DC, the Alliance is "dedicated to promoting regulatory, research, and reimbursement policies that will foster innovation in regenerative medicine" in addition to serving as a "source of information about regenerative medicine for policy makers, the media, and the general public".
With Atala's 'encouragement', Michael Wener of DC law firm Holland & Knight and Morrie Ruffin of Adjuvant Global Advisors signed up to be the driving force in the creation and evelopment of the Alliance intented to bring together academic institutions, non-profit organziations, and companies that commit to supporting and participating in a lobbying organization to promote much-needed policies in support of the regenerative medicine sector.
The organization intends to announce its final list of charter members later this month. Thus far its a respectable list including Wake Forest Institute for Regenerative Medicine, Stanford University, the University of Washington, Georgia Tech University, the Genetics Policy Institute, Geron, Johnson & Johnson, Aldagen, iZumi, Fate Therapeutics, Maxcyte, Kleiner, Perkins, Caufield and Byers, and Proteus Ventures.
Werner and Ruffin both have a great deal of experience with Capitol Hill and the biotechnology sector. I had a great conversation with Werner this week and am very encouraged by the plans for the Alliance and their intended inclusiveness. In my opinion, the Alliance promises to fill a much-needed gap unmet by any existing organizations. I will dedicate a post to more information about the Alliance in the days to come.
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FINANCIAL
Surprisingly, the past week or so - here in the beginning of summer with July 4th weekend being most everyone's primary focus in America - was a very big week for closing financing deals in the cell therapy sector.
In this financial environment the ability to raise any money is a marked accomplishment. Nevertheless four companies in this emerging field did just that: a technology company, a service-based one, a company developing a therapeutic, and one that sort of does all three.
Celsense, Inc. announced they recently raised just over $1.76 million in additional funds in their Series B.
The funds will reportedly be used to commence human clinical testing of its lead compound, Cell Sense which is a fluorocarbon tracer agent used to label cells ex vivo without the use of transfection agents. The technology reportedly enables researchers and clinicians to non-invasively track the administration and migration of the labeled and transplanted therapeutic and/or diagnostic cells using 19F MRI or MRS. Applications include tracking cells in immunotherapy or regenerative medicine as well as diagnosis of inflammatory sites by tracking selected populations of immune cells.
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NeoStem, Inc. (NYSE Amex: NBS) has also bucked the trend recently by tapping into Asian investors and deal-making. NBS announced that it now has raised additional gross funds of over $4 million from institutional and private investors. These funds, together with the $11 million private placement from three Asia-based investors announced April 13, 2009, bring the total investment obtained by NeoStem in the past two and half months to over $15 million.
The funds are said to be tagged for sundry purposes including support of the company's continuing initiatives with their VSEL (very small embryonic-like) stem cell technology licensed from the University of Louisville, advance NeoStem's expansion activities in China, expand U.S.-based operations, add seasoned management to the NeoStem team with experience in clinical drug development in pharmaceutical and biotechnology industry, fund the administration of overseas based clinical trials, and other general corporate purposes. Suddenly that $15 million doesn't seem like nearly enough for a focus spread that broadly
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Neuralstem, Inc. (NYSE Amex:CUR) announced that an investor fund has purchased $1 million worth of stock and purchased $3 million worth of warrants to be exercised in $1 million allotments to be exercised within a year, three years, five years from the date of issuance. Neuralstem plans to use these proceeds for "working capital" much of which - according to SEC-filings - is currently being spent on legal fees.
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International Stem Cell Corporation (OTCBB:ISCO) entered into a definitive agreement for a $5 million financing investment commitment (the “Investment”) with a biotechnology-focused fund (the “Investor”) that has offices in New York and California. The Company may draw down funds from the Investor as it finds a need, but is not obligated to do so. Funds will be drawn down through the issuance of redeemable Series E Preferred Stock.
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I usually don't track or include reports of grants being received by companies but this one is a good size and - I admit - more importantly I wanted to point you to their unique usage of social media.
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Pluristem Therapeutics Inc. (NasdaqCM:PSTI) (DAX:PJT) has been awarded a $1.9 million government grant from the Office of the Chief Scientist (OCS) at the Ministry of Industry, Trade and Labor of Israel, as a government participation in R&D expenses for the period March 2009 to February 2010. The OCS awards grants to industry in Israel to foster technological innovations. This is the fourth consecutive year that Pluristem has received this grant. The funds will be designated and used by Pluristem to support the clinical trials of the allogeneic placental-derived adherent stromal cell product, termed PLX-PAD, for the treatment of critical limb ischemia (CLI), the end-stage of peripheral artery disease (PAD), as well as for other research and development activities of the Company.
Here's the cool part: Check out their product animation on YouTube.
CLINICAL
Ok, this isn't exactly a clinical story but it's the closest thing I've got this week and I hate leaving sections bare.
Geron Corporation (Nasdaq: GERN) has published in advance of print in the journal Regenerative Medicine a paper that reports that dendritic cells (DCs) scalably manufactured from human embryonic stem cells (hESCs) exhibit normal functions of naturally occurring human DCs found in the bloodstream. This, they say, supports their planned use of hESC-derived DCs in their therapeutic vaccine currently in Phased II for AML but employing autologous dendritic cells. (see GEN's summary here)
COMMERCIAL
The big deal of the week, of course, was between GE Healthcare, a unit of General Electric Company (NYSE: GE), and Geron Corporation (Nasdaq: GERN).
The companies announced an agreement to co-develop human embryonic stem cell (hESC)-derived assay products for in vitro drug screening with the aim of having products on the market in by "early 2010".
Under the worldwide, exclusive license and collaboration agreement, GE Healthcare obtained an exclusive license to Geron’s hESC IP and a sublicense under Geron’s rights to foundation hESC patients held by the Wisconsin Alumni Research Foundation. IP arising from the alliance will be shared between the companies, with GE Healthcare retaining rights to the resulting technologies for drug discovery applications and Geron for cell therapy applications.
When Geron announced that its IND had been accepted by the FDA earlier this year, Okarma made quite a bit about how they had figured out how to scale the production of their product " unlike adult stem cell therapies" and that their product was "scalable in the same way as a monoclonal antibody".
This theme continues in this announcement with Geron's David Earp saying "“Geron is intensely focused on developing hESC-based cell therapies, and the expertise that we have developed in scalable manufacturing and differentiation of hESCs to specific cell types is directly applicable to the production of these dells for drug discovery.”
Interestingly, GE claims it is bringing "expertise in cell manufacturing" to the deal. GE Healthcare will fund the R&D program and will be responsible for manufacturing, sales and distribution of products developed under the agreement.
Click here for more background information
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As has been discussed here several times, ever since Cell Genesys Inc (NASDAQ: CEGE) pulled the plug on its late-stage trials of its GVAX immunotherapy triggering Takeda Pharmaceuticals to pull out of its co-development deal for the product, Cell Genesys has been busy dealing with downsizing, lawsuits, and shopping itself around to potential suitors looking primarily to take it on for its cash.
This week BioSante Pharmaceuticals, Inc. (NASDAQ: BPAX) anted up to announce it is merging with Cell Genesys in an all-stock transaction valued at about $38 million - pretty much the exact amount of cash CEGE has in its bank account. the surviving entity will be BioSante.
Cash value is actually a generous purchase price for biotech companies which usually come with a burn rate that makes then worth less than their cash. CEGE, however has no clinical program, had sold most of its assets, and had downsized to 9 staff in a rented office.
There are, of course, persistent rumors of back alley shenanigans that took place in CEGE's demise but we'll leave these on the street. What is interesting to me is that BioSante has suggested they will examine options for the future of Cell Genesys' GVAX Immunotherapies, including potential combination with BioVant, BioSante's vaccine adjuvant, as well as possible external collaborations. The combined entity is also on record saying they will try to outlicense other Cell Genesys technologies.
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Pantheon China Acquisition Corp. ("Pantheon") (OTCBB:PCQC) announced that its shareholders approved its plan to purchase China Cord Blood Services Corporation ("CCBS"). Pantheon has changed its name to China Cord Blood Corporation and will continue to trade on the OTCBB under the symbol PCQC until its expected transition to a larger U.S. exchange is complete. Pantheon China Acquisition Corp. is a "special purpose acquisition company" formed for the purpose of acquiring businesses in the People's Republic of China. Pantheon China consummated its initial public offering on December 20, 2006 generating an aggregate gross proceeds of $34,500,000. China Cord Blood Corporation is currently the largest cord blood banking operator in China in terms of geographical coverage and is the only market player with two (i.e., Beijing and Guangdong) out of six exclusive cord blood bank licenses issued by the PRC government authorities to date. Under the current PRC government regulations, only one licensed cord blood bank operator is permitted to operate in each licensed region.
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Former CEO, Ed Scott, retired late last year from Lifeblood Biological Services, LLC with a plan to buy it out. Scott has now successfully closed that transaction and has rebranded as Key Biologics, LLC. The beauty here is that the company has a steadily growing book of business since its 2005 insception and Scott bought the assets of LBS for $188,000 on previous 12-month revenues of close to $1.6 million.
Key Biologics is being positioned to service the burgeoning cell therapy market. For insight into just some of the market need the company is poised to address, click here to read an article by Scott, previously published a year ago.
The company produces nearly 300 products for researchers involved in cellular-based therapies. Among other types of products and custom services, Key Biologics provides researchers with products made from human blood collected at its office which it converts into customer-specific products like leukocytes, plasma or T-cells shippint them directly to its customers per their specification.
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Living Cell Technologies Limited ASX:LCT; OTCQX:LVCLY) announced that it has now officially opened its new designated pathogen free pig breeding facility in New Zealand to support the imminent launch of its clinical trial in New Zealand of DIABECELL in subjects with type 1 diabes. DIABECELL is an encapsulated porcine insulin-producing cells which can be administered without the need to use immunosuppressive drugs.
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GEN this week carries an interesting article on a company applying its existing technology to cell therapy bioprocessing scale-up. The article sites "...large-scale culturing of embryonic and induced pluripotent stem (ES, iPS) cell lines for use in regenerative medicine and drug discovery applications" as still a "major challenge" for the industry. Cyntellect, Inc. reports that its Stem Cell Manager "is a series of stem cell management processes including automated physical passage of stem cells, automated embryoid body generation, and purification of specialized cell types derived from stem cells" which can help alleviate some of the major challenges in large-scale automation of the processes required to manipulate sensitive therapeutic cells.
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Bio-Matrix Scientific Group, Inc. (OTCBB:BMSN) announced the launch of a national marketing program aimed at hospitals "in need of storing umbilical cord blood specimens for future transplantation".
The business model is not entirely clear to me but it would appear BMSN believes hospital clients will outsource the processing and storage of cord blood units which parents donate to the hospital-owned public cord blood "bank" (or registry). I'm told BMSN may not have the appropriate license for processing of this sort but I'm sure they will be taking care of that. More importantly, I am keenly aware this hospital-based model for selling back-end cord blood processing and storage to hospitals is a very tough sell and not one that hospitals understand easily. For what it's worth, that didn't stop the WallStreetCorner.com from naming the company its “Stock Pick” for the second time since February of this year.
We will watch BMSN's future with interest.
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Toronto-based bio-commercialization center, MaRS Innovation, has selected an umbilical cord stem cell technology as its first commercialization opportunity. The technology offers a proprietary method to create multi-potent stem cells (MPSCs) from human umbilical cord blood with several promising therapeutic applications but which they will initially focus on diabetes. Pre-clinical data has reportedly demonstrated that these cells uniquely secrete insulin in response to glucose, thereby mimicking the “normal” physiological state.
MISCELLANIA
ProChon Biotech, Ltd., a company working with tissue regenerative technologies announced that it recently received the Genzyme Award for Excellence in Cartilage Research for its poster presentation entitled, “Comparative Analysis of Cell Sources for Cartilage Cell Therapy.” The company reports that the research recognized by the award is incorporated into their development pipeline and will compliment its BioCart™ Cartilage Regeneration System.
Prochon's BioCart™ Cartilage Regeneration System is an autologous chondrocyte implant system that - and here's why I love this story because this is just another example of a commercially-available cell therapy that people in the US never talk about - is commercially available in Italy, Greece and Israel and is undergoing an FDA Phase II multicenter clinical study in the United States. The company reports that to-date over 70 patients have received the BioCartTM implant with some patients over five years post-implantation.
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I came across a nice piece this week discussing Arteriocyte Medical Systems' work on its DARPA-funded project to create human blood from stem cells at a rate that could allow the military and hospitals to produce units of blood on site. The company claims that with a technology developed by a company called Nanex, they are able to grow stem cells at a rate 10 to 100 times faster than with more traditional methods. The Nanex technology mimics a bone marrow environment in which stem cells can grow quickly. The trick now is to automate and scale-up the system to meet the specs DARPA required in its project paramaters.
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Luke Timmerman, a recent Twitter convert (@ldtimmerman), and one of the most prodigious reporters on the biotech scene, has posted another great article on Dendreon with this great opening line: "Dendreon, as part of its plan to morph into a full-fledged company that discovers, develops, and markets cancer drugs, has worked behind the scenes to transform its shareholder base from a Wild West gallery into a roster of backers you might expect from a blue-chip biotech." The article, "Dendreon Tames Short Sellers, Transforms Itself into Old-School Buy-and-Hold Stock", is - as usual - a good read.
**
The LA Times carried a very thorough and informative piece on the state (promise and challenges) of therapeutic cancer vaccines that is also well worth the read.
**
Another interesting article I uncovered this week was a rather poorly written but informative review of some the scientific, clinical and regulatory progress in the field of stem cell research/therapy in India.
Sign-off
The latest issue of the journal Regenerative Medicine has been released and it looks well worth the read. Here the table of contents.
Hope this was useful.
This week I'm ROTFLMAO loving this cartoon so I thought I'd share the chuckles (thanks to www.onstartups.com). I can't tell you how many times in the past years I've seen th
e inevitable 'hockey stick' market projections graph from companies, analysts, and investors when discussing the coming 5 years in the cell therapy sector. It's become a standing joke about how we just keep pushing the hockey stick out to the right but the curve is inevitably the same. This pokes a little fun at this much maligned tradition.
Maybe it's just because I'm Canadian that I love this so much but c'mon the beloved hockey stick has become almost more a marketing tool than the venerable piece of sports equipment every Canadian boy must own! Maybe we can adapt this new hockey stick model to give the over-used Gartner cycle a run for its money!
Down to business...
I was told last week that Senators have been inviting people to Capital Hill to speak to them about FDA regulation of adult stem cells perhaps at the instigation of Chris Centeno's lobbying through ASCTA (www.stemcelldocs.com) that the medical use of moderately expanded autologous adult stem cells should not be regulated by the FDA but rather self-regulated by physicians. This could, of course, have serious implications on business models for autologous cell therapy companies. I've heard nothing of the Senators asking for ISSCR or ISCT counsel to-date. It would, of course, likely be Centeno's position that anyone involved in those orgs are "pharma hacks" as he has accused me of being.
ASCTA which is comprised of physicians none of whom are acknowledged leaders in stem cell research recently proudly announced the release of their stem cell guidelines which they suggest should replace FDA regulation. In what appears to be simple ignorance of the years of work being done by AABB & FACT, ASCTA described their guidelines as "the world's first clinical guidelines to allow U.S. doctors to safely begin using the patient's own stem cells to treat disease." Their lab practice guidelines contain such gems as: - Any facility used in the processing A-ASC’s must be of suitable size, construction, and location to prevent contamination.
- The facility should be in a good state of repair.
- Equipment used in the facility should be adequately maintained
- In humid areas, dehumidifiers should be used to control the spread of infectious agents in the lab.
- Any reagents used must be approved for human use (where practical).
In an online posting entitled "The FDA is Killing Adult Stem Cell Therapy Thus Killing Patients", ASCTA supporter, Don Margolis, states:
In a startling new interview on stem cell research and adult stem cells in the near future, Dr. Christopher Centeno warns that Big Pharma and the FDA are teaming to control the use of a person’s own stem cells thus slowing innovation and the use of Adult Stem Cells to help patients now.
FINANCIAL
ReNeuron Group plc (LSE: RENE.L) has raised the £3 million it announced it intended to raise. ReNeuron recently received regulatory approval to commence a Phase I clinical trial in the UK with its lead ReN001 stem cell therapy for disabled stroke patients. The Company is developing stem cell therapies for a number of other conditions, including peripheral arterial disease and diseases of the retina. ReNeuron has also developed a range of stem cell lines for non-therapeutic applications – its ReNcell® products for use in academic and commercial research. The Company’s ReNcell®CX and ReNcell®VM neural cell lines are marketed worldwide under license by USA-based Millipore Corporation.
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Pfizer expects to pump $100 million into its international stem cell development program which aims to focus both on small molecules that work by modifying cells in the body and cell therapy where the cells are manipulated outside the body. The first major use of the Pfizer money is for a collaboration with University College London (UCL) for the treatment of certain forms of blindness through the London Project to Cure Blindness. The collaboration will attempt to develop stem cell-based therapies primarily for wet and dry macular degeneration (AMD), which Pfizer has the rights to progress through clinical trials and then commercialize. Pfizer’s work on this could potentially provide a boost for the cash-strapped and searching-for-a-buyer, Intercytex Group plc (AIM: ICX), whose involvement on the London Project could yield cash royalties. But that's not where the Pfizer-Intercytex connection may end, apparently. Reports indicate that Pfizer is in fact eyeing the troubled Intercytex as a potential acquisition or investment. The company is on the auction block and actively seeking suitors. Cytori Therapeutics, Inc. (NASDAQ: CYTX) expected to close a private placement for $4.2 million by May 11. No word yet...
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Cord Blood America, Inc. (OTCBB:CBAI) has received a commitment for up to $2.3 million of new funding to acquire or build its own state-of-the-art laboratory for the storage of multiple stem cell products including umbilical cord blood stem cells. Less than two years ago the company signed a deal with Progenitor Cell Therapy LLC to process and store their stem cell units on a contract basis. Now CBAI says having their own facility will over the long term reduce their costs for both the processing and storage and additionally allow CBAI the flexibility and capacity to pursue new sources of revenue such as the storage of adipose tissue and peripheral blood stem cells processing and storage much easier than under the current structure of outsourced relationships.
CLINICAL
Aastrom Biosciences, Inc. (Nasdaq:ASTM) temporarily suspended enrollment and patient treatment in its U.S. Phase II IMPACT-DCM clinical trial following a report that a patient died at home after being released from the hospital following treatment in the trial. IMPACT-DCM is a clinical trial to evaluate the surgical delivery of autologous cells directly into the human heart muscle for the treatment of congestive heart failure associated with dilated cardiomyopathy (DCM) in both ischemic and non-ischemic patients. The patient's cause of death has not yet been determined and is the subject of a pending investigation at the clinical site. An independent Data Safety Monitoring Board (DSMB) will also assess the circumstances of the event. The Company has voluntarily suspended patient enrollment and treatment in the trial and the FDA placed the trial on temporary clinical hold pending an investigation.
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ImmunoCellular Therapeutics, Ltd. (OTC: IMUC.OB ) (IMUC) announced that clinical data from the company's Phase 1 trial of its antigen-pulsed dendritic cell immunotherapeutic cancer vaccine ICT-107 for patients with brain stem glioma and glioblastoma will be presented at this year's ASCO. Having said that, they also said that while "encouraging data" was observed from the phase I trial of ICT-107, IMUC's primary focus going forward will be on its lead product candidate, ICT-121, which is an peptide-based "off-the-shelf" cancer vaccine that targets cancer stem cells and may have applicability to multiple types of cancer. IMUC anticipates filing an Investigational New Drug (IND) application in the third quarter of 2009 for a Phase I trial of ICT 121 in the treatment of glioblastoma (brain cancer).
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Little-known, Memgen, LLC will be presenting results from a recently completed phase I clinical trial of its novel active immunotherapy product, ISF35, in patients with high-risk, progressive chronic lymphocytic leukemia (CLL), at next week's ASCO meeting. According to the study's principle investigator, Dr. Castro, one injection with no chemotherapy resulted in durable reductions in lymph nodes and spleen size in most patients and three of fifteen patients with rapidly progressing disease did not require any further treatment for more than one year. Based on the results from this single injection study, a phase II trial evaluating multiple intranodal injections of ISF35 in CLL has begun funded by - of all things! - the Food and Drug Administration at UCSD. The company believes the product is also positioned for use in lymphomas and solid tumor cancers.
**
Aderans Research Institute Inc. (ARI) announced it has launched Phase 2 of its clinical study on cell-based hair regeneration for men and women. The company's research is focused on developing a state-of-the-art tissue engineering solution to the regeneration of hair, taking actual hair cells--fibroblasts and keratinocytes, the two primary cell types within hair follicles--and promoting their growth in controlled laboratory environments. The research teams then recombine them and these "combined hair cells" are then placed in the patient's skin, where they are expected to elicit hair growth.
**
Cytori Therapeutics, Inc. (NASDAQ:CYTX) completed enrollment in the first study to investigate adipose derived stem and regenerative cells in chronic heart disease. The trial, named the PRECISE study, was designed as a double-blind, randomized, placebo controlled, dose escalation study specifically enrolling patients suffering from an advanced form of chronic heart disease, known as chronic myocardial ischemia, for which there is no generally accepted treatment. The trial enrolled 27 patients in a study involving the patients’ own cells extracted from adipose tissue and processed for delivery at the point of care using Cytori’s Celution® System. The cells were then injected back into the patients using the NOGA® XP System (Biologics Delivery Systems, Cordis Corp., a Johnson and Johnson company). Primary endpoints were safety and feasibility of Cytori’s Celution® System as part of a novel procedure for chronic heart disease.
A major attribute of the Celution® output is that it is comprised of multiple cells types. This, the company believe, contributes to multiple mechanisms of action, and thus increases the potential to restore heart function. The Celution® System used in PRECISE was configured specifically for cardiovascular disease, including proprietary processes and enzymes needed to achieve a level of purity required for vascular delivery.
COMMERCIAL
Cytori Therapeutics (NASDAQ:CYTX) and GE Healthcare, a unit of General Electric Company (NYSE:GE) announced an agreement by which GE Healthcare will commercialize Cytori's StemSource(R) technology in the North American stem cell banking and research markets. The StemSource technology includes automated equipment to process stem and regenerative cells found in adipose tissue, cryopreserve them or use them directly for research purposes. In January 2009, Cytori and GE Healthcare formed a separate agreement to commercialize Cytori's products in ten European countries. This includes selling the Celution(R) 800/CRS System in the European cosmetic and reconstructive surgery market as well as selling StemSource products in the European cell banking and research markets. The recent partnership is similar in nature to the European agreement, but is limited to the sale of StemSource banking and research products in the U.S., Canada and Mexico for 18 months starting in the second quarter of 2009. The agreement does not include U.S. commercialization of Cytori's Celution System, which is currently under review by the FDA.
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Celsense, Inc. has announced the formal opening of its new and expanded R&D facility said to be a key component to Celsense’s commercialization strategy for its pre-clinical and clinical-grade molecular imaging agents used to non-invasively visualize and measure cellular activity and biodistribution. The company is now actively bringing both their lead research and clinical-grade cellular and molecular imaging agents to market over the coming months and is said to be collaboration with several academic and industry parties currently evaluating our products for potential use in their discovery research or clinical protocols.
Their clinical-grade Cell Sense reagent is a fluorocarbon tracer agent used to label cells in vitro then transplanted thus enabling investigators and clinicians to non-invasively track the administration and delivery of therapeutic and diagnostic cells in vivo using MRI. Applications include tracking therapeutic cells in regenerative medicine and immunotherapy applications, and observing localized immune system response by tracking populations of immune system cells. The research-grade V-Sense is an injectable fluorocarbon MRI tracer agent that labels leukocytes in situ, enabling the direct, non-invasive observation of immune system response and localized inflammation. Applications include observing change in immune system response to therapeutics and mapping localized disease such as certain cancers and infections.
**
After securing a $12M equity financing facility and closing a $1.5M private placement, formerly on-the-rocks Melbourne-based Prima BioMed (ASX: PRR), which had less than $500,000 in the bank at the end of December, is now preparing IND filing in the US for a phase IIb / III pivotal trial of CVac for ovarian cancer. As previously reported here, the company has also commenced CVac™ ovarian cancer treatments on selected patients in Australia made possible through the Australian Government’s Australian Regulatory Control Mechanism’s Special Access Scheme under the Therapeutic Goods Administration. Meanwhile the company is looking at taking the product to other countries as a means of generating cash flows in the short-term through the sale of CVac™ treatments. Now the company's executive director, Mr Martin Rogers, is touring North America in discussions with a number of hedge funds and other biotech focused investment funds about new investment and partnering opportunities for the commercialization of CVac.
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Progenitor Cell Therapy, LLC has done a deal with NeoStem, Inc. (NYSE Amex: NBS) as their exclusive provider of commercial adult stem cells processing and storage. Progenitor founder, Andrew L. Pecora, has also joined NeoStem's Advisory Board.
**
NeoStem, meanwhile, - the new favorite company of alternative "medical" products promoter, Suzanne Sommers - has been busy the last few weeks creating a curious empire of sundry types of bedfellows. Recently the company announced it had signed an agreement to license the exclusive worldwide rights to a technology, with pending patent applications, developed by Vincent Falanga, M.D., Chairman of the Department of Dermatology and Skin Surgery at Roger Williams Medical Center, Providence, R.I. Dr. Falanga's stem cell product under development, Primcel, is a mesenchymal stem cell (MSC) product. Early clinical studies are said to indicate that Primcel can be used to accelerate or jump start healing of chronic wounds. Dr. Falanga's work seeks to develop a prepackaged product created from a patient's own cells, ready for physician use in a clinical setting, consisting of three applications over a 12-week period.
Almost in the same breath, the company filed a patent application claiming the proprietary stem cell technology of Vincent C. Giampapa, M.D., F.A.C.S. relating to cosmetic facial rejuvenation, which NeoStem first licensed in February 2009. Dr. Giampapa is director of the Giampapa Institute for Anti-Aging Medical Therapy, a board-certified plastic reconstructive surgeon and Assistant Clinical Professor of Plastic and Reconstructive Surgery at the University of Medicine and Dentistry of New Jersey. His cosmetic stem cell face lift technology is said to comprise a "non-surgical procedure for complete facial rejuvenation that involves injecting pluripotent cells, including stem cells, into the skin of individuals whose skin has lost its firmness and texture due to age".
Days later NeoStem announced it had signed an exclusive royalty-bearing license agreement for the Asia territory for a procedure developed by Regenerative Sciences, LLC and marketed for the treatment of chronic orthopedic conditions, under the name, Regenexx. Additionally, Regenerative Sciences, through its founder and CEO, Christopher Centeno, M.D., will serve as a consultant to NeoStem, Inc. in the area of stem cell therapy in orthopedics.
Undoubtedly Centeno will also embroil and leverage NeoStem in his fight with the FDA about the marketing of Regenexx in direct flagrance of FDA regulation. In any event this is clearly a solid "plan B" for Centeno should the FDA ever get around to shutting him down in the U.S. In the interim, Centeno continues to strengthen his network of supporters and is reportedly actively working Capital Hill for support for his fight.
To mix it up a little, NeoStem then announced they had signed a deal to promote and glean royalties from the sale of a liquid, nutritional supplement - AIO Premium Cellular Health. Not kidding. They have signed an agreement for Ceres Living, Inc., a developer and direct marketer of health and wellness products. AIO Premium Cellular Health was apparently developed in conjunction with NeoStem's scientists and Advisory Board members, based on certain nutraceuticals that have been shown to "optimize stem cell functions". The promotion of AIO is said to be "part of NeoStem's corporate commitment to advancing anti-aging and regenerative medicine therapies and technologies."
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Novocell, Inc. announced that it has received U.S. Patent # 7,534,608 with with wide-ranging method claims covering the Company’s innovative stem cell therapy for the production of functional pancreatic, insulin-producing cells from human embryonic stem cells (hES). Novocell’s therapy is being developed as a method for the use of hES cells to replace insulin-producing pancreatic cells that are destroyed in people with diabetes.
**
Neuralstem, Inc. (NYSEAmex:CUR) recently published a reaction to a press release by StemCells, Inc. (STEM) refuting many of their statements in what has become a very public dispute. Included in the reaction is the claim that "the PTO upheld the patentability of Neuralstem’s core technology in May, 2006, in response to a challenge from STEM" and that the Neuralstem "patents are not being challenged in the PTO or in any of the suits with STEM".
**
This is not a cell therapy story but it could be. Takeda Pharmaceutical Company Limited (TSE: 4502) and IDM Pharma, Inc. (Nasdaq: IDMI) today announced that Takeda America Holdings, Inc., a wholly-owned subsidiary of Takeda (Takeda America), and IDM Pharma have entered into an agreement for Takeda America to acquire IDM Pharma. Takeda America has established Jade Subsidiary Corporation as a wholly-owned subsidiary to effect that transaction. Under the agreement, Takeda America will purchase all of IDM Pharma's outstanding shares for US$2.64 per share in an all cash tender offer followed by a merger.
You may recall that on April 1 last year (omen perhaps?), Cell Genesys, Inc. (“Cell Genesys”, Nasdaq: CEGE) and Takeda announced that the companies had formed a global alliance for the development and commercialization of GVAX immunotherapy for prostate cancer, Cell Genesys' lead product candidate then in Phase 3 clinical development. Under the agreement, in exchange for exclusive worldwide commercial rights to GVAX immunotherapy for prostate cancer, Takeda was to pay Cell Genesys an upfront payment of $50 million and additional milestone payments totaling up to $270 million relating to regulatory approval and commercialization of GVAX immunotherapy for prostate cancer in the United States, European Union and Japan.
This deal quickly unraveled when they decided to mothball both phase 3 studies of GVAX on statistical likelihood of failure. This was Takeda's big venture into cell therapy and it is an ugly, recent memory. Now they have bought IDM Pharma. They 've bought it for the company's primary asset, MEPACT, (mifamurtide), a macrophage activator drug therapy indicated for the treatment of non-metastatic osteosarcoma (malignant bone cancer) following surgical removal of the tumor (resection) in children, adolescents and young adults.
Here's the cell therapy part: IDM Pharma has 3 autologous cell therapy products currently on developmental hold as a means of perserving resources to get them to the home run on MEPACT. BEXIDEM is activated macrophages (Monocyte-derived Activated Killer cells or MAK® cells) for bladder cancer. UVIDEM, for melanoma, and COLLIDEM, for colorectal cancer, are both therapies using dendritophages (specialized immune cells derived from the patient's own white blood cells). It will be very interesting to see what Takeda does with these products, if anything.
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Veterinary stem cell company, Vet-Stem, Inc, has now expanded from its US base into Canada, providing commercially available autologous, adipose-derived stem cell tranplants for horses and dogs. **
According to the Seattle Times which played a critical role in exposing what is now alleged to be potential fraud, CellCyte Genetics, "left a cashless zombie after the stock-touting campaign that lifted its value to $400 million faded away, says it has reached a tentative settlement in the Securities and Exchange Commission (SEC) inquiry into its activities" but it still faces a criminal probe and the "number of lawyers embroiled in the company's legal troubles have now outnumbered the company's peak work force".
MISCELLANEA
International Stem Cell Corporation (OTCBB:ISCO), the company behind human stem cells from unfertilized eggs (called “parthenogenetic stem cells”), submitted comments to NIH on their draft stem cell research guidelines issued by the National Institutes of Health (NIH) on April 17, 2009. ISCO then published its comments in a press release and then their CEO published a blog to address questions regarding their comments.
Genetics Policy Institute also submitted a letter to the National Institutes of Health (NIH) commenting on the Draft Guidelines for Human Stem Cell Research. The complete letter is available at www.genpol.org for public viewing.
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The California Institute for Regenerative Medicine is working its way through Congress seeking $500 million in federal funds it hopes to apply toward guaranteeing the long-planned small-business loan program.
Forbes published what is actually quite an informative article on cell therapy medical tourism for a change.
Sign-off
If you've read this far you're obviously interested in the cell therapy industry. If you're on LinkedIn make sure you have joined the LinkedIn Cell Therapy Industry Group.
Thank you all for all your good wishes about the new addition to my family - my own little sample of regenerative medicine! Now I gotta go and spend some time with her before I'm enrolling her in college...
Remember. Cell Therapy Means Business.
Here we are in April 2009. The much newly anticipated date when we expect the Dendreon saga to head toward a climax. Few people know the Dendreon story better then Xconomy journalist, Luke Timmerman who does an outstanding job of summarizing the saga and stakes in Dendreon's immiment Provenge gamble. Indeed tomorrow is the beginning of what we now anticipate will be the final chapter in this epic sage. Tomorrow (at 9am EDT), Dendreon will discuss the final results from their phase III IMPACT trial of Provenge for prostate cancer (call details here).
Here are a couple random bits to kick us off:
___________________________________________________________________
This issue of blog is sponsored by the 'next advance' in innovative, reliable and low-cost laboratory instruments.
You wanna feel the rush of getting quality lab products for lower prices? You wanna rush to NextAdvance.com.
Not settling with organizing doctors against the FDA with his "American Stem Cell Therapy Association (ASCTA)" (see my previous blog comments), Dr. Christopher "the renegade" Centeno is now inciting patients to oppose the "FDA's position that the patient's own adult stem cells are drugs and should be regulated as such." (Click here for the press release.)
To facilitate patient activism he has launched a patient web-site, safestemcells.org, to drive the activities and opinions of patients in what he's calling the Safe Stem Cells NOW! movement. The Stem Cells Now! movement's goal? "To inform patients and physicians that their ability to access safe stem cell treatment is being heavily restricted by pharmaceutical industry agendas and by the FDA."
He has also recruited the enthusiasm of Barbara Hanson, co-founder of stemcellpioneers.com, who is quoted as saying:
"Adult stem cells are cells from our own body. They are very safe. There are no moral or ethical issues. They are safer than taking aspirin and yet the FDA has classified our own stem cells as drugs that require regulation. This means that prolonged investigations, including lengthy clinical trials, will be required for each and every disease and application that adult stem cells could be used for. This could take years and years. It smells of big pharma to me and many others."
For my other posts about the work of Dr. Centeno, see Regenexx vs the FDA 2009 and Cell therapy is not the practice of medicine.
FINANCIAL
Some people I talk to in the sector say they feel weirdly immune from the entire economic meltdown because the industry seems to be ticking along just fine in many ways continuing to be fueled by its own internal optimism and external enthusiasm for the promise of cell-based therapies...well that, and the still-growing amount of money being spent on the research side of the sector. Others, are certainly feeling the pinch. The dividing line may well be those who are trying to raise money and those who are not.
Having said that, here we go with reports of money being raised -- all from non-US investors mind you. Proving that its economy is not down and out, the first two report of fundraises come from Down Under.
There's no way I can start this first story any better then The Australian did in its March 12 report (that I only now just picked up now): "From the miracles-do-happen file comes this provident yarn of a $3.4 million market cap tiddler that has secured $12 million of funding for further development of its ovarian cancer drug, CVac."
They're talking, of course, about Prima BioMed Limited (ASX:PRR) which has been busy reinventing itself with new management and money since Biomira took a pass on its option to partner on CVac development more than a year ago. The company has been slowly bleeding money and management since. The company is still clearly on a low burn because I had to mine this information from anywhere but direct sources. The company itself has been very quiet about any of this.
The news is this: Prima Biomed has secured access to a $12-million funding facility from investment bank Fortrend Securities to help commercialize the company’s ovarian cancer treatment. The deal allows Prima to place shares with Fortrend as needed over the next three years.
Prima said it will use the cash to move through a phase IIb trial and towards a pivotal US registration trial of its dendritic cell-based ovarian cancer therapy, which the company says is designed to stimulate the body’s own immune system to attack cancer cells. The company said it now hopes to begin a US trial by mid-year, after the company reported positive feedback on its pre-investigational new drug meeting with the US Food and Drug Administration last year.
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Not to be outdone by the magic tricks of its pennystock sibling, the much more stable Mesoblast Limited (ASX: MSB) which reported on 31 December 2008 having $9.6 million of capital at its disposal announced that it has successfully raised an additional $10.81 million by completing a private placement to existing, as well as new, institutional and sophisticated investors. The new funds will be used to expand the company’s clinical trial programs focusing on bone and cartilage regenerative products for spinal vertebral disc disease. These programs will be pursued in parallel to the Company’s ongoing Phase 2 clinical trial in knee osteoarthritis. The placement of 15.02 million shares was reportedly oversubscribed and was made at a 10% discount to the closing price of the Company’s shares on 25 March 2009.
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NeoStem, Inc. (Amex: NBS) reports they have completed an $11 Million series D private placement financing from three Asia-based investors, including a private equity firm operating in partnership with strategic investors. Investors are RimAsia Capital Partners, LP, a pan-Asia private equity firm operating in partnership with a regional network of strategic investors drawn from leading Asian families and companies, investing $5 million; Enhance Biomedical Holding Corporation based in Shanghai, and investing $5 million and Elancrest Investments Ltd., a Singapore-based firm, investing $1 million.
While NeoStem makes mention of using the funds in various ways including the development of its VSEL (very small embryonic-like stem cells) technology licensed from the University of Louisville, what they really seem focused on is expansion activities in China, including those relating to its pending acquisitions and medical tourism - defined as travel by people whose primary and explicit purpose is to access in a foreign country medical treatment not yet available in their own nation.
NeoStem expects to connect U.S. citizens with advanced therapies not yet available in the U.S., and attract people from other countries to seek regenerative therapies in China. Indeed Eric Wei, Managing Partner of RimAsia Capital Partners, LP is quoted as saying, "We are extremely optimistic about the growth opportunities for NeoStem based on its multi-pronged plan to capitalize on a growing PRC-based network. We are glad to be joined by other investors in Asia who recognize the huge market potential for stem cell therapies in the PRC [People's Republic of China], a field in which breakthroughs are developing at an accelerating rate."
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In a UK report about how stem cell innovation is at risk, the study authors from The University of Nottingham, funded by the Engineering and Physical Sciences Research Council (EPSRC), reports that the global cell therapy industry, reports that the global cell therapy industry:
"...now involves nearly 200 companies developing primary and secondary cell therapies, plus another 180 banking cord blood. In total the global cell therapy industry currently has sales of over $1 billion a year and a steady number of products are now reaching late stage clinical trials."
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Crystal Research Associates, LLC has issued an Executive Informational Overview® (EIO®) on Neuralstem, Inc. (CUR: NYSE-Alt)
CLINICAL
Amorcyte, Inc recently presented data at the American College of Cardiology (ACC) Annual Scientific Session from its Phase I clinical trial of AMR-001, the company's lead product for the treatment of damaged heart muscle following acute myocardial infarction (AMI), that showed a significant relationship between cell dose and biologic effect. According to the company this is the first and only study to prospectively define a dose of a purified and potent autologous stem cell therapy (AMR-001) that resulted in a significant improvement in perfusion, a trend towards improved cardiac function and the potential to reduce subsequent adverse cardiac events following AMI. Emory University - one of the study sites - also issued its own press release on the data.
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At the same meeting, TCA Cellular Therapy’s Medical Director Gabriel Lasala, M.D. presented preliminary results of an adult stem cell treatment for severe limb ischemia from its Phase I safety/efficacy clinical trial using a combination of the patient’s own endothelial progenitor cells (EPCs) and mesenchymyal stem cells (MSCs) obtained through bone marrow aspiration then mixed and infused into damaged veins.
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MolMed S.p.A. (MLM.MI) announced that results obtained in the European multicentric Phase I/II study (TK007) of its TK therapy have been published in the April issue of the prestigious medical journal The Lancet Oncology. The article - Infusion of suicide gene-engineered donor lymphocytes after family haploidentical hemopoietic transplantation for leukemia: the TK007 phase I/II trial - gives insight into the outcome of the trial, conducted on 54 adult patients, that resulted in significant survival improvement of patients affected by high risk acute leukaemia receiving bone marrow transplants from a partially compatible family donor (haplo-transplants). In this context, the introduction of TK therapy allows the use of add-backs of donor T lymphocytes, that promote a rapid and wide immune reconstitution, abating transplant-related mortality and permitting long-term survival.
These trial results were, of course, already communicated to the market and included in MolMed’s Offering Circular and allowed MolMed to get authorisation to begin a Phase III randomised trial that started in Italy in Spring 2008, and will be expanded to other European clinical centres this year. The Phase III trial TK008 is pivotal for the registration of TK therapy, that could become among the very first cell therapies using genetically engineered cells to obtain marketing approval. MolMed’s partner for the Asian markets, Takara Bio Inc. (OTCPK:TKBIF), started clinical development of TK in Japan in October 2008, with a Phase I trial in relapsed leukaemia patients conducted at the National Cancer Center in Tokyo.
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These breasts and slim waist are brought to you by Cytori Therapeutics, Inc. (NASDAQ: CYTX) and GE Healthcare. Available soon to women for only £6,500?
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While Prima BioMed has been closing the deal on its new financing it has also apparently obtained permission from the Australian TGA's Special Access to treat a patient with its CVac ovarian cancer treatment. The injection of the vaccine in ovarian cancer patients works as a postsurgery and post-chemotherapy maintenance therapy to delay relapse and control metastases. While the company is currently not in clinical trial in Australia and has not yet filed its IND for FDA approval in the U.S., the company has sought special exemption for a single-use treatment for what must be a very special patient.
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Osiris Therapeutics, Inc. (NASDAQ:OSIR) announced the treatment of the first patient in a Phase II clinical trial evaluating Prochymal for the treatment of heart attacks.
COMMERCIAL
Further to my speculative post here last week, we've now learned that ULURU Inc. (NYSE Amex: ULU) has signed a non-binding offer letter to acquire York Pharma plc ("York"). York Pharma is a United Kingdom based skin care company with operations or distribution networks throughout Europe with an established revenue base. By way of reminder, the reason this is of interest here is that York has two cell therapy products on the UK market for the treatment of chronic wounds and burns: Myskin™ and Cryoskin™.
While the companies have not yet executed definitive documentation relating to any acquisition transaction and there's no guarantee they will, Uluru is hoping potential benefits will significantly accelerate the maturation of ULURU into a commercially based company with a strong revenue base and positively position the company for more rapid growth both in the United States and Europe.ULURU Inc. is a specialty pharmaceutical company focused on the development of a portfolio of wound management and oral care products to provide patients and consumers with improved clinical outcomes through controlled delivery utilizing its innovative Nanoflex(TM) Aggregate technology and transmucosal delivery system
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Novocell, Inc. announced that it has received U.S. Patent 7,510,876 with claims covering human definitive endoderm cells, an essential cell for generating not only pancreatic type cells, which Novocell is developing for use as a cell therapy for diabetes, but also other endoderm lineage-derived tissues and organs such as lungs, intestine, liver, thymus and thyroid.
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Progenitor Cell Therapy LLC board member Robert A. Hamm promoted as COO of drug maker Biogen Idec.
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Looks like WuXi PharmaTech Incorporated has some serious buyer's remorse of it $162.7 million purchase of AppTec Laboratory Services Inc.
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Advanced Cell Technology, Inc. (OTCBB: ACTC.PK) could receive up to $1.9 million in fees as a result of a new licensing agreement with its Korean partner. ACT has agreed to license certain retinal pigment stem cell technology to CHA Bio & Diostech Co. Ltd. for development and commercialization exclusively in Korea. The two companies have a recently formed joint venture called "Stem Cell & Regenerative Medicine International" located in Worcester, MA and focused on the development of human blood cells. It is majority owned by CHA Bio and benefits from ACT exclusively licensing all of its hemangioblast technology to the joint venture.
If all milestones are met in this new collaboration focused on stem cell technology to treat a variety of eye diseases, ACT stands to earn $1.9 million in fees. CHA will pay all fees associated with clinical trials expected to take place in Korea. Meanwhile, ACT has applied to the U.S. Food and Drug Administration to begin clinical testing of its retinal pigment stem cell technology during the second half of the year.
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Presumably to help soften the financial blow of its interrupted phase III trial of Prochymal for Crohn's disease, Osiris Therapeutics, Inc. (NASDAQ:OSIR) has announced that it has agreed with NuVasive, Inc. (NASDAQ:NUVA) to accelerate transfer of the Osteocel business. Under the terms of amendments to the existing agreements between the parties, NuVasive will assume responsibility for Osteocel processing by April 10, 2009 and has removed contingencies applicable to $30 million of the $45 million in remaining milestone payments.
Osiris is now scheduled to receive $30 million in cash or NuVasive common stock in 3 schedule payments all before the end of September. The terms applicable to the remaining $15 million milestone payment are unchanged. This additional payment becomes due and payable when NuVasive achieves $35 million in cumulative Osteocel sales. NuVasis has projected $29 million in Osteocel sales in 2009.
To date Osiris has received from NuVasive $40 million in upfront and milestone payments. Effective April 10, Osiris will cease all operations associated with Osteocel processing. Concurrent with the amendments to the Asset Purchase and Manufacturing agreements, Osiris will transfer its Osteocel inventory to AlloSource, of Centennial, Co., which had been processing quantities of Osteocel for Osiris and has now agreed to produce Osteocel solely for NuVasive. NuVasive has entered into a separate outsourcing supply agreement with AlloSource for the continued manufacture of Osteocel.
Because of that deal, they are now able to layoff 80 people.
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It looks like the ever well-tanned Burton Feinerman is back in control of his company Stem Cell Biotherapy which he at least appeared to lose temporarily to his former partner Casey Nabavi until Nabavi abandoned ship late last year to start a competing stemcell transplant company, Cellulogix. Now it looks like things are going to bet rough between the former partners. Stem Cell Biotherapy ("SCB") has filed suit in Los Angeles County Superior Court against the Company's Co-Founder and former President Casey Nabavi of Calabasas, California, for among other things conduct by Nabavi giving rise to patient claims against Nabavi that he had misrepresented himself as a medical doctor, practiced medicine without a license, supervised the injection of stem cells which caused some patients to become ill and personally advised patients how to manage these side effects. Nabavi/Cellulogix have offices/clinics in Los Angeles, Tampa, Montreal, Zona Rio (Mexico) and Istanbul. The SCB lawsuit also seeks to recover SCB funds in excess of $1.65 million that Navabi paid himself before leaving SCB, to recover assets including intellectual property belonging to SCB which Navabi took and used to start-up the new Cellulogix business, to prevent Nabavi from continuing to compete with SCB in contravention of the parties' non-competition agreement and for SCB to recover ownership, the business and profits of Cellulogix as belonging to SCB not to Navabi as a result of Nabavi's wrongful conduct and activities. A copy of the SCB lawsuit against Nabavi and Cellulogix can be viewed at the Company's web site www.stemcellbiotherapy.com.
SCB is in the business of "assisting persons who desire to try stem cell therapy treatment for serious medical problems which have not responded to traditional medical treatments." SCB uses medical clinics and specialized doctors in locations around the world where stem cell therapy is authorized to treat medical conditions such as spinal cord injury, ALS, Parkinsonism, Alzheimer's, multiple sclerosis, autism, cerebral palsy, brain damage, heart disease, COPD, kidney diseases, eye diseases, diabetes, transverse myelitis, etc.
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Adding themselves to the growing list of companies looking to leverage their cell-based technologies to get to market early with some type of non-therapeutic low-hanging fuit in order to finance their more serious, therpeutic product development aspirations, Stem Cell Therapy International Inc. (OTCBB: SCII) -- soon to be known as AmStem International after a successful merger with Histostem Ltd, of South Korea -- has decided that its first product will be a "cosmetic face cream made with stem cells" to be sold wholesale to major cosmetic distributors worldwide. This is expected to provide AmStem with an influx of capital to finance "expansion in other areas".
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Proving there may never be an end to the types of stem cell banking models that continue to pop up, StemSave™, Inc. is now encouraging dental patients to bite on their offer to bank stem cells collected from their disposed teeth. StemSave’s patented technology turns routine trips to the dentist into "potentially life-saving experiences".
So we can now pay to save our stem cells from our umbilical cord blood, umbilical cords, amniotic fluid, placenta, adipose (fat) tissue, menstrual blood, bone marrow, blood (after stem cell mobilization or not), and teeth. Hmmm.
RESOURCES, EVENTS & MISCELLANEA
Regenerative Medicine Vol. 4, No. 2, March 2009 is now available online.
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Stem cell scientist Dr. Robert Lanza, Chief Scientific Officer of Advanced Cell Technology has managed to find time away from changing the world of science and modern medicine to pen a book with astronomer Bob Berman on Biocentrism , a subject that challenges our traditional, outdated perceptions on life, time & space, and even death.
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The New York Academy of Sciences, China’s Ministry of Health, the Chinese Academy of Medical Sciences, the Chinese Academy of Sciences and international partners including the Medical Research Council of the UK and the German Research Foundation are collaborating to host a conference entitled ‘Regenerative Medicine’, May 15-16, 2009 in Beijing, China. The meeting is intended to foster collaborations between industry, academia, government, and finance with a world-renowned scientific organizing committee including Nobel Laureate Dr. Torsten Wiesel and the Chinese Minister of Health, Zhu Chen. The scientific program includes Drs. Anthony Atala, John Gearheart, and Stefanie Dimmeler, among many other leaders from industry, academia, and the clinic. A full conference agenda can be found at www.nyas.org/regmed
Sign-off
That's all I got.
Thanks again to lab equipment supplier, Next Advance, for supporting this issue of the Cell Therapy Industry Hilites. They found me on Twitter, you can too. I'm @celltherapy.
