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Monday, September 28, 2009

The true status of the cell therapy industry...

I apologize for the quality of this image but hopefully its just clear enough for you to make out this week's Genetic Engineering News poll. If you're reading this in realtime you will see on GEN's homepage.

In case you can't read it, the poll is this:

"Which type of stem cells will be the first to move from the laboratory into clinical testing?
  • embryonic stem cells
  • induced pluripotent stem cells
  • adult stem cells
  • none of the above
  • undecided"
The introduction to the poll is as follows:

"The 2009 Lasker Awards honored two scientists who developed nuclear reprogramming, a process that instructs specialized adult cells to form pluripotent stem cells. This has been widely hailed in the life science research community and the biotech/biopharma industry as one of the most significant findings of the past decade. While embryonic stem cell research has been plagued by ethical concerns, some believe that the newfound ability to induce pluripotency in adult stem cells will lead to breakthroughs in the therapeutic applications of stem cells. So we want to know which type of stem cells you think will win the race."

C'mon GEN, are you serious? If you honestly missed the point that adult stem cells are already in clinical testing and use, then shame on you. If you're trying to catch people in their own ignorance by this trick question then I suppose that's a little more tolerable but a poll like this still perpetuates the misinformation - at least until you do everything you can to expose it.

This kind of misunderstanding about the true status of the development of cell therapies is pervasive. Late last year I commented on an article by Tom Feilden on BBC's website in which he said things like,
  • "...a sizeable number of exciting stem cell projects are now reaching the stage where they should be moving on from the research lab and into clinical trials" and
  • "Regenerative medicine may finally be moving out of the lab and into the clinic."
As we all know, stem cell research moved from the lab into clinical use decades ago (with stem cell transplantation) and there are now literally hundreds of clinical trials currently underway testing the use of different kinds of stem cell products for almost every imaginable condition not the least of which is Osiris' Prochymal which has been tested now in two phase III clinical trials.

Perhaps the educated readers of this blog will think that it is not possible that readers of GEN could believe that adult stem cells are not already in clinical testing. Let me assure you there is a lot of misinformation about the maturity of the cell therapy sector even among those in biotechnology. As proof, a click on "View Results" shows that as of today the poll stands almost neck-and-neck. A mere 48.6% of respondents believe adult stem cells will beat ESC or iPS cells to clinical testing. 45.7% believe either ESC or iPS cells will beat adult stem cells into clinical trial.

What more can I say? We - you and I - must continue to do everything we can to raise awareness about the true status of the cell therapy industry and the products we are developing - even among our biotech colleagues.


p.s. I know my Cell Therapy Industry HiLites has disappeared this summer. I'm doing everything I can do resurrect it from among the rubble left by summer holidays, home renos, new baby, and more work from clients than I could have ever expected.

Monday, July 20, 2009

Cell Therapy Industry HiLites 2009-07-17

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.

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.


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.
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".


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).


Bioheart, Inc., (OTCBB: BHRT) has raised approximately $1 million over the past few month in an ongoing offering (see details above).
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
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.
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.
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.
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.


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.
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.


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.
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.
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.
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.
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.
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
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.
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.


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:
Check out Daniel Kraft's TED talk on his MarrowMiner technology being developed by StemCor Systems, Inc.
After 15 years in the biotech conference industry, the Williamsburg BioProcessing Foundation has shuttered its operation in financial distress. Hasta la vista, Wilbio.


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.


Thursday, July 9, 2009

SBIR Funding - Should it be open to VCs?

I don't have much time to give you an extensive blog about the issue but I did want to bring this to your attention. Here's the nutshell.

The NIH's SBIR (Small Business Innovation Research Program) funding mechanism has been an important one for small businesses who are not big/mature/saavy/desperate-enough to land venture capital funding, have very limited access to other NIH funding, and have tapped out the available angel/seed/friends_n_family resources at their disposal.

Since 2003, one of the hallmarks of the rather small SBIR funding pot ($2.2 billion a year) has been that it was not available to companies that are majority-backed by venture capital. Before 2003, venture-backed firms were allowed to participate in the SBIR program.

The program is scheduled to expire next month and so is up for renewal. The House committee responsible for the program has proposed that the VC-restriction be removed thus re-opening the program to a whole slew of companies previously ineligible. The House just passed this bill. A senate version of the bill tries to reach a compromise by limiting the amount available to venture-backed firms.

Whatever your opinion on the judiciousness of this move, there is no doubt that opening up eligibility to the program will dilute the available funding.

Here is a link to yesterday's article on the subject in the Washington Post.

Fellow blogger Steven S. Clark (BioScience Biz) has a just posted his most recent update and opinion on the matter that is well worth the read.

Friday, July 3, 2009

Cell Therapy Industry HiLites 2009-07-03

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.


More stakeholders united in one place at one time than any other.


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.
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
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.
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.
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.
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.


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)


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
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.
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.
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.
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.
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.
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 from naming the company its “Stock Pick” for the second time since February of this year.

We will watch BMSN's future with interest.
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.


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.
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.

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.


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.

Friday, June 26, 2009

Cell Therapy Industry HiLites 2009-06-26

Let's get the public services announcement out of the way right up front.
  • Curiously, the Regenerative Medicine Foundation (I know. I hadn't heard of it either until now.) has announced that its "Translational Issues in Regenerative Medicine" conference is the "premiere event for leaders in regenerative medicine translation, representing academic research, clinical, healthcare policy, regulatory, reimbursement, investment and biotechnology industry interests." Curious because it has yet to ever occur. That being said, if they do what they promise, it may just well be. I'm trying to learn more and when I do I'll let you know. Meantime, this is about all I know:
Spring 2010
Twin City Quarter
Winston-Salem, NC

" facilitate business development opportunities, the conference will feature a venture forum including 15 regenerative medicine companies, as well as partnering sessions."

Do yourself a favor. Stop monkeying around with home-made cryopreservation brew or, for than matter, any media not built to minimize cellular damage from freezing and thawing.

Do your cells a favor. Try CryoStore and HypoThermosol.

I'm dedicating this issue of Cell Therapy Industry HiLites to
TiGenix NV (NYSE EURONEXT: TIG). TiGenix wins the Cell Therapy Group "kick-ass cell therapy company of the week" award which is, of course, something I just made up but they had have a great week raising several million dollars, announcing plans for a new European facility, and receiving a positive opinion from the EMEA CHMP which all but assures them of an MAA within 90 days applicable in 30 European countries. See details below.


has secured the necessary financing to fund the set-up of a GMP manufacturing facility in Europe through a private placement for a total amount of EUR 5,4 million. The investment agreement was subscribed by NV Industriebank LIOF, Particon BV, Limburg Ventures BV and LRM NV. The funds will be used to set up a new cell expansion facility in Europe for the commercial production of its cell based products in the Netherlands. The financing for the facility has been secured through a private placement. After carefully evaluating a number of options throughout Europe, taking into consideration technical, logistical, regulatory and financial criteria, TiGenix has selected a building on the Chemelot Campus, near to Maastricht, the Netherlands, to locate its new cell expansion facility. The site is centrally located in TiGenix’ key European markets, in a region that is strong in distribution and (bio)logistics and that is highly committed to develop as a transnational knowledge centre in life sciences and regenerative medicine.

This financing has now been completed following the execution of a long term lease agreement for the facility in question.
After raising $40+ million last year and over $14 million so far this year, Cytori Therapeutics Inc (NASDAQ: CYTX) has announced it has entered into an agreement to raise up to another $20 million over the next year. The company announced recently that it had raised $850,000 in what is described as an agreement by Orlando, FL-based fund, Seaside 88, to buy 7.15 million shares over the next year by buying a specified allotment every two weeks at an agreed discount of the then current stock price. The agreement could being in roughly $20 million for the company depending on share price. Cytori intends to use the proceeds to "accelerate marketing efforts and continue our investment in clinical trials to broaden the number of potential applications for which the Celution® System may be applied".
A bankruptcy court hearing is scheduled for July 6 in which Isolagen Inc. (AMEX:ILE) will request final approval of its debtor-in-possession financing plan under Chapter 11 bankruptcy reorganization protection.
Always a master at the art of spinning good profile, NeoStem, Inc. (NYSE: NBS) (AMEX: NBS), in the business of collection, processing and long-term storage of adult stem cells for future medical need, garnered a feature in Gene Marcial's prominent Business Week column, "Inside Wall Street," on June 18, 2009. The same week saw the company profiled in a piece on the investment potential for adult stem cell businesses in a Fortune Magazine article distributed by The company has been on a deal-making, press release and publications tear the past few months. "Big plans by tiny NeoStem (NBS)," Mr. Marcial observed, "have helped catapult its stock to $2.27 a share from 50 cents on March 9." It must be the plans because the company which has been doing what it does since 2006, generated a mere $45,100 in total revenuefor the three months ended March 31, 2009. The good news is that is up from the $700 generated in the three months ended March 31, 2008.

The company also announced another deal saying it has signed an exclusive 10-year agreement with Enhance BioMedical Holdings Limited, a Shanghai corporation, to develop a Stem Cell Collection and Treatment Network using NeoStem's "technologies" Taiwan, Shanghai and the Chinese provinces of Jiangsu, Zhejiang, Fujian, Anhui and Jiangxi. Enhance BioMedical Holdings is a subsidiary of Enhance Holding Corporation, a multinational conglomerate whose CEO, Jackson Ling, recently invested $5 million in an $11-million private placement financing for NeoStem.


Osiris Therapeutics, Inc. (NASDAQ:OSIR) announced six-month interim data from a Phase II clinical trial evaluating Prochymal, the Company's formulation of adult mesenchymal stem cells, for the treatment of chronic obstructive pulmonary disease (COPD). The trial failed to demonstrate an improvement in pulmonary function in COPD patients. The company, quickly becoming practised at spinning what might be considered bad news, said they were pleased the data did provide additional evidence of safety for the product in addition to demonstrating significantly decreased inflammation in the COPD patients. Sixty-two patients were enrolled and are being followed for two years in the placebo-controlled study.
Pluristem Therapeutics Inc. (NasdaqCM:PSTI) (DAX: PJT) received approval from the Paul Ehrlich Institute to begin clinical trials evaluating the company’s placental-derived adherent stromal cell product, PLX-PAD, for the treatment of critical limb ischemia.
Aastrom Biosciences’ (NASDAQ:ASTM) trial of its cell therapy for severe chronic heart failure was released from a clinical hold by the FDA. The the agency determined the therapy did not cause the death of a participant which resulted in the hold in May.
The University of Pittsburgh Cancer Institute began enrolling patients in a Phase III study evaluating the safety and efficacy of Gamida Cell Ltd's StemEx cord blood stem cell therapy for the treatment of blood cancers.
Having received regulatory approval late last year to proceed with its clinical trial in New Zealand, Living Cell Technologies Limited (ASX:LCT; OTCQX:LVCLY) CEO Paul Tan said enrolment of patients should be complete in about two months to begin testing their pig islet cells implanted in humans to treat diabetes.
Neuralstem, Inc. (NYSE Amex: CUR) has entered into a sponsored research agreement with the China Medical University & Hospital of Taiwan, in Taichung, Taiwan, to prepare for a human clinical trial using Neuralstem’s human spinal cord neural stem cells to treat stroke patients. The therapy will focus on patients whose post-stroke symptoms, including complete or partial paralysis, have stopped improving more than six months after an ischemic stroke.


Not to be outdone by finance and operational teams, the regulatory team at TiGenix also announced reason to celebrate this week. TiGenix (NYSE EURONEXT: TIG) announced that the company received a positive opinion from the Committee for Advanced Therapies (CAT) and the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMEA) on the European Marketing Authorisation application (MAA) for
its lead product ChondroCelect.

CHMP opinions are forwarded to the European Commission for adoption of the final community Marketing Authorisation, which is typically issued between 60 and 90 days from adoption of the CHMP opinion. I'm told that European Commission generally follows the advice of the CHMP but, as Dendreon, there's little reason to celebrate until the formal approval is formalized. For the EMEA take and related info on the decision, click here.

Upon approval, ChondroCelect will be, I believe, the first product approved under the new ATMP regulatory framework. It will certainly be the first cell based product to be centrally approved in the 27 member states of the European Union as well as in Iceland, Lichtenstein and Norway under the new Advanced-Therapy Medicinal Products (ATMP) legislation.
Further to my March 31 speculation about who might be the buyer of York Pharma plc (and its two cell therapy products on the market) and my April 13 post that ULURU Inc. (NYSE Amex: ULU) had signed a non-binding offer letter to acquire the company, ULURU Inc. (NYSE Amex: ULU) has announced that at this time discussions to acquire York Pharma, plc have been terminated. ULURU has notified York that the $1 million USD line of credit it had extended York are due plus accrud interest. The revolving credit facility is secured by substantially all of the assets of York and its subsidiaries. York is now actively pursuing alternative funding facilities in order to repay the line of credit within 30 days and to meet its ongoing working capital requirements.

Meanwhile, rumor has it the company is already in talks with another potential suitor.
Here are a couple hints for your next press release. It's rarely newsworthy to have as the primary point of your release an announcement about what you are going to do, rather than what you've done; secondly, don't announce seemingly contradictory information. I'm just saying....

Stem Cell Therapy International, Inc. (OTCBB: SCII), through its wholly owned subsidiary AmStem International, Inc., announced that it has "started the process" to enable it distribute Histostem's products in anticipation of the finalization of the merger between Stem Cell Therapy International, Inc. and Histostem. This is intented, according to the SCTI President and CEO, to "enable AmStem International, Inc. to begin US distribution and immediately bolster the company's cash flow". However, the product has yet to be the subject of a clinical study in the U.S. for which they are seeking "investigators...of the highest caliber, to compare its Stem Cell Facial Cream with others on the market."

The companies reportedly continue "to work on the necessary steps to finalize the merger, and both sides are working diligently to manage its many details."
Community Blood Services has entered into a licensing agreement with AuxoCell Laboratories, Inc. to use AuxoCell’s exclusive patent rights and propriety process to extract fetal mesenchymal stem cells from the Wharton’s Jelly of donated umbilical cords, allowing Community Blood Services to operate the first Wharton’s Jelly derived stem cell bank in the United States.
After netting $221 million last month by nearly 12 million shares at $19.20 apiece, Dendreon Therapeutics Inc (NASDAQ:DNDN) expects to spend up to $50 million in a two-phase expansion of its manufacturing facility in Morris Plains, New Jersey. Monday in a regulatory filing. Additional quality control laboratories, data center, training areas, infrastructure and offices are scheduled to by done by mid-December. The final phase, with additional manufacturing clean-room work stations, production support areas, warehouse, infrastructure and offices, is to be substantially complete by April 23 of next year. Dendreon finished the initial build-out of its 158,000-square-foot facility in July 2006, before its FDA application was tabled to await additional clinical results.
Apparently yet another way has been invented to derive stem cells from placenta - reportedly a source much richer in stem cells than umbilical cord blood. The inventors are looking for commercial partners.
Cell Genesys, Inc. (NASDAQ:CEGE) - the company that just won't go away - is still pursuing "strategic alternatives". Meanwhile, it expects its cash balance will be approximately $36 million upon imminent completetion of its stock exchange offer.


This week's issue of Science has a special focus on stem cells including a piece on "" by Donald W. Fink, Jr. of FDA, CBER, an article on stem cell tourism and 3 reviews.
26 June 2009. Vol 324, Issue 5935, Pages 1603-1754
The Production Assistance for Cellular Therapies Group (PACT) Educational Web Seminar: Topic: "Deviation Management of Type 351 & 361 Cell Products" Date: Thursday, July 16, 2009 Time: 12:00pm-1:00pm (Eastern US Time) Register is now open for this Web Seminar at


Thanks again to BioLife Solutons. Congratulations to Tigenix. May the force be with all of you and for those going to ISSCR later this travels and please try not to enjoy Barcelona too much without me!

Tuesday, June 23, 2009

A Therapeutic Long Tail. Does Cell Therapy Fit the Model? (Part II)

...this is a continuation of Part I...

So are Personalized Medicines the Long Tail of the Therapeutics Industry?

In 2006, Chris Anderson, Editor-in-Chief of Wired magazine, released "The Long Tail" – a book describing the Web 2.0-induced business-to-consumer economics (r)evolution enabling businesses to now deliver product at outrageously affordable prices to global micro-markets.[i] The book quickly became one of the most influential business books of the ‘new economy’.

Indeed this flat new world – as Thomas L. Friedman describes it[ii] – is all about micro-trends and micro-markets, empowering small companies and niche products to participate successfully in the marketplace along with Fortune 100 companies and blockbuster products. More than that, this paradigm shift is forcing product and service companies to revolutionize their business and pricing models, the latest and rabidly successful of which is the pricing model of “Free!”. The blockbuster-based business model that depends on the economics of a ‘hit’ (whether it’s a platinum-selling CD or a ‘blockbuster’ drug) is no longer seen to be the only way to make money and, indeed, may not have any future at all.

To date, this internet-driven Long Tail (r)evolution has had its largest impact on – if not been largely restricted to – the retail sector. Business-to-consumer (B2C) companies are now enabled like never before to profitably supply unique global products to meet customer demands. In this respect, the (r)evolution has had a visible impact on the retail side of the pharmaceutical and biotech industries. However the ongoing realization of “personalized products” as the future of medicine for consumers seems a perfect fit for the Long Tail economic model.

But does this apply to therapeutics?

To the extent that the Long Tail economy is less about the mass market and more about businesses succeeding in serving niche markets with unique and/or customized products, the analogy fits.

Certainly, it would appear the sun is setting on the "blockbuster hit" days of the therapeutics industry. In June 2008 Genentech's Susan Desmond-Hellmann, president of product development, when outlining the biotech giant's development strategy to a group at the Goldman Sachs healthcare conference, told the group that the blockbuster drugs of the past may never be duplicated. “Future drugs,” she stated, “will cater to a smaller, better defined group of patients” and “the days of big cholesterol-lowering drugs for millions of people may be behind us.” (link).

In a June 8, 2009 post, authors of the IN VIVO Blog stated that pharma "managers these days are learning to leverage only very modest top-line growth into faster bottom-line growth through cost-savings, bolt-on acquisitions, and rapid introductions of incrementally-improved new products. The traditional, high-risk, high-growth R&D-based model is gone, in case you hadn't noticed."

The second indication that personalized medicine fits with the Long Tail supply-demand model is that therapeutics can now be customized taking advantage of personalizing elements such as HLA-typing and advances in genetic, diagnostic, and discovery testing that will permit us to more effectively both pre-select which medicines are best suited for which patient sub-groups and develop therapies that are better suited to particular genetic groups. On the clinical end of the spectrum, advances in theragnostic tools and surrogate biomarkers (e.g., imaging) are allowing clinicians to much more rapidly assess personal responses to treatment and adjust their therapeutic regimines accordingly before any symptomtic response can be observed.

The ability to match therapeutics to genetic make-up, disease stage/presentation, or predisposition will allow the provision of treatments with more confidence in their expected efficacy, fewer patient-specific adverse reactions, and result in cost savings to the health care industry and a better return to fund the premium for the development of such products.

In a recent interview with Xconomy's Luke Timmerman, Alan Frazier, founder and managing partner of Seattle-based Frazier Healthcare Ventures, one of the world’s biggest life sciences venture capital funds, said,

The trend is toward smart pharmaceuticals that address smaller populations, narrower indications, with better results. It’s based on a better understanding of disease from genomics and proteomics. You’ll see more targeted drug delivery. You’ll have incredibly focused pharmaceuticals, which will take care of some of the safety issues you read about.

Does the model apply to cell therapies?

At the intersection of the personalized and regenerative medicine sectors, lies the therapeutic industry’s response to (or reflection of) both the demand for and the ability to deliver a more personalized therapeutic to the consumer (the patient).

If the tail of the therapeutics market is being stretched longer to include products better targeted to smaller markets, this therapeutics Long Tail will almost certainly include regenerative medicine (regen 2.0) products and many of these will be cell-based therapies.

The cell therapy sector now encompasses the convergence of three distinct technologies, i.e. cell transplantation, tissue engineering, and biomaterials. Often also drawn in are elements of gene and molecular (e.g., proteins and antibodies) therapies.

Autologous cell therapies are the ultimate personalized medicine and while such medicines are often not included in definitions of personalized medicine, any approach to regenerative medicine – whether using tissue engineered products, cell-based therapies, small-molecule drugs, scaffolds, nano or biobots, etc – intended to trigger in vivo regeneration or repair... is personal!

Again, to the extent the Long Tail economy is about “selling less of more” then medicines such as cell therapies will certainly be part of the long tail of the therapeutics industry giving access to highly-personalized, tailored products to meet the unique needs of the consumer.

But does the Long Tail economic model apply?

While there may well be blockbuster cell therapies in the future, concern around the scarcity of potential blockbusters in cell therapy are seated in the notion that the blockbuster economy is the only successful model.

Who thought you could make money by giving free access to internet searches and email? There are a lot of ways the analogy breaks down but what the Long Tail economy has shown us is modern corporate ingenuity in finding profitable business models in niche markets despite the long-standing dominance of blockbuster products.

Where the paradigm falls short, in my opinion, is when one realizes that a fundamental pillar to the Long Tail economy is the commoditization of products and production processes as well as the standardization of delivery systems. The Long Tail economic model is enabled by the fact that the cost of goods sold has been brought sufficiently low enough - by new technologies which allow for small batches to be produced and shipped - that the products in question are affordable to most. Highly unique and/or customized goods were always available to those with sufficient ability to pay. The Long Tail brings these kinds of products to micro-market at the same price or lower than their mass-market counterparts.

We are a long way from this kind commoditization of personalized medicines and, in particular, of cell-based therapies. There is little-to-no standardization of production systems at any scale. The next generation of these therapies will still be highly priced goods which many people, insurers, and/or healthcare systems will be pressed to afford.

In once sense, this is not surprising. Even in the world of electronic or digial products, Moore's principle only applies because the cost of goods for new technologies when they first enter the market are prohibiitively expensive. If the model applies in the therapeutics industry, technological advances in production will bring down the cost of goods and therfore the price.

On the other hand, what drives the price of therapeutics has not been so much the cost of goods (production) but the need to recoup massive upfront R&D costs which must be recovered (with a reasonable profit) during the life of a patent.

Will this change with personlized medicines and cell therapies? One could argue it might if personlized medicines and cell therapies can deliver on the promise to have a much higher effective rate among those given the therapies. Pharmaceuticals have a notoriously low effective rate among those prescribed any given drug. New research methodologies employing surrogate biomarkers, imaging, cell-based assays, etc promise the potential to significantly lower R&D costs by (a) getting to a much quicker "go/no-go" decision point in therapeutic development, and (b) identifiying patient sub-groups for which the therapeutic will have a high rate of effectiveness. One the one hand, this has the potential to lower the cost of goods (driven by R&D costs) and simultaneously justify a higher price point because of a high rate of therapeutic success.

However, even if we were able to materially lower upfront R&D costs, this still does not change the fact that cell therapies are costly to produce on a per-unit basis and the opportunity to "scale-up" is limited particlarly with autologous cell therapies which require lot sizes to be an individual batch. While this is a seperate discussion, I am confident technological and/or commercial innovation will find a way to economically bring therapeutics to market that are proven effective. I am not alone. Recenly Howard Liang, MBA, Ph.D., Analyst and Biotechnology Managing Direct at Leerink Swan as quoted as saying that “Autologous manufacturing is not the deal breaker it used to be. If these [cell-based] vaccines work for really difficult-to-treat diseases, the manufacturing issues will be worked out.” ("Special Report: Customized Cancer Vaccines Finally (Maybe) Arrive". GEN News Highlights. 12 June 2009)

There are other reasons the Long Tail business model as it is currently defined does not fit cookie-cutter with the therapeutics industry not the least of which is that the consumer demand does not solely regulate supply. In highly regulated industries, the economies are different. Some would argue this should not be the case particularly if personalized medicines, genomics,theragnostics, etc advance to point where the consumer has an abundance of therapeutic information and options but that is the subject of another discussion.

In the end, it is certainly clear to me that the emerging technologies are enabling new therapeutic paradigms that will introduce elements of the Long Tail business model into the therapeutics industry.

How much Impact will Cell Therapies Have on New Therapeutic Paradigms and Systems?

Despite the exponential escalation of cell therapy innovation, is cell therapy ready to be a viable business that will make any material impact on the therapeutic industry? For reasons which I will continue to explore in this blog in the months to come, I believe the answer is 'yes'.

The cell therapy industry has more products in late-stage development than many believe. While the existing commercial products are far from blockbusters and questions remain regarding the business models and potential margins for cell therapy products, ground-breaking research and market enthusiasm is driving a renewed interest among an early second-round of investors including participation by mature industry players making cautious plays in the sector. Regulatory issues in the primary markets of the US and EU are rapidly becoming less problematic though harmonization remains a significant challenge.

In blogs to come I will outline the commercial developments I see transpiring that make me believe the primary issues around the 'business models' question are being addressed. Regenerative and personalized medicines are enough of a force that creative business people will (a) invent ways to lower the cost of goods and (b) figure out how to price and reimburse appropriately to ensure effective therapies get to people that need them and incentivize doctors to prescribe them.

Most indicia point to this sector being poised for a phase that will start demonstrating real answers to those challenges which have to-date prevented many from participating in a sector that they believe presents too many unanswered risks.


As with so many of the paradigm-shifting economic revolutions in the past, once the underlying technologies exist, business and economic models radically adjust to reflect and accommodate that change. The latest of these is the Long Tail economy (r)evolution with it ability to deliver niche (if not personalized) products to niche markets. Pesonalized medicines - regenerative medicine and cell therapies in particular – are the therapeutic industry’s answer for the demand for personalized products. For a host of reasons, however, the economic model is not yet one which makes for profitable delivery of such products to niche markets.

Nonetheless, while questions about the viability of the business and economic models for these paradigm-shifting therapies are reasonable, the next 5 years will show that today’s questions about the business models for cell therapy were rooted in limited imagination about the potential for technological and commercial innovation as well as outdated concepts of the therapeutics industry and its economies.


[i] Anderson C: The Long Tail: Why the Future of Business is Selling Less of More. Hyperion (2006).

[ii] Friedman TL: The World is Flat. A Brief History of the Twenty-First Century. Farrar, Straus and Giroux (2005).