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Another > $100M month for companies in the cell therapy space

By Dr. Matthew Watson

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Last month we reported here on this blog that March was more than a $100M month for companies in the stem cell and cell-based regenerative medicine space in terms of monies raised.  

What we missed was a $15M grant from Cancer Prevention and Research Institute of Texas (CPRIT) for UK-based CellMedica.  This pumps last month's total to just under $140M.

This month, according to our sources, betters even March's better numbers by coming in at just over $170M though that is largely on the back of one large deal in Asia.  Here's how the numbers break down.

Allocure kicked off the month with a decent $25M Series B round from new syndicate member Lundbeckfond Ventures, as well as previous investors SV Life Sciences and Novo A/S.  Allocure is headed into phase 2 for acute kidney injury with an allogeneic mesenchymal stem cell therapeutic they currently call AC607.  


Little-known Canadian-based, Sernova then announced a $3.6M PIPE to fund continued development of its proprietary Cell Pouch System(TM), and, in particular, to fund the upcoming first-in-man clinical trial for patients with diabetes receiving an islet transplant.  The application to proceed with this trial is currently under review by Health Canada.


Next up was NeoStem closing a $6.8M public offering for "expanding" their contract manufacturing business, Progenitor Cell Therapy, and "enrolling the PreSERVE AMR-001 Phase 2 clinical trial for preserving heart function after a heart attack".  


The biggest deal of the month was a $65M convertible debt financing of China Cord Blood by none other than global powerhouse Kohlberg Kravis Roberts (KKR) through it KKR China Growth Fund L.P., a China-focused investment fund managed by KKR.  We believe this is deal is certainly an investment in the future of China's healthcare market potential but that it is bigger than that.  We believe a significant driver for this deal may likely have been the opportunity to consolidate this sector globally - to use a significant operation and 'war chest' to fund mergers and acquisitions on both the public and private cord blood banking sector worldwide.


The only classic first-round venture raise this month was a milestone-based $5M Series A by Bay City Capital into Phil Coelho's new company, SynGen, to fund his latest iteration of stem cell processing devices.


Forbion Capital then announced that it was leading a series D round, joined by fellow existing investors TVM Capital, Lumira Capital, Intersouth Partners, Caisse de depot et placement du Quebec, Morningside Group, and Aurora Funds, of $25M into Argos Therapeutics in order to kick them into their phase 3.  The hope here is that with some early phase 3 data they may be able to attract the elusive partner they couldn't land with a mere bucket of phase 2 data.


Innovacell landed the only European deal by announcing an 8.3M Euro (~$11M) investment by Buschier, Fides, HYBAG, and Uni Venture.  This will be used for the continued clinical development of its cell-therapy (ICES13) for the treatment of stress-urinary incontinence currently in a ph 3 study in several European countries.


ReNeuron announced a private placement also open to existing shareholders that brought in just under $10M (£6.1M) to support their phase 1 trial in stroke and other pre-clinical, clinical, and regulatory milestones. 


Finally, the Bio-Matrix Scientific Group, in an apparent ongoing quest to continuously reinvent itself, announced at month's end that they had formed a new subsidiary named Regen BioPharma and that they had raised $20M in a financing commitment from Southridge Partners II to purchase its common stock as required over the term of the agreement at a price set by an agreed formula.  This money is said to be dedicated to the acquisition of discovery-stage intellectual property and driving it through to phase 2 trials in an exercise of maximum value creation over a period they claim to be as short as 18-24 months.


..


So in the end, the month saw companies in the space raise just over $170M and even if you back out the stem cell banking deal its still over $100M for cell therapy companies.  


Over the 2 months, then, we've seen just over $311M raised through a variety of means by companies at every stage of maturity and for intended purposes ranging from acquisition, consolidation, early stage clinical development, and phase 3 testing.


--Lee


p.s. If you are aware of other deals in the sector this month, let us know and we'll update this accordingly.


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To Read More: Another > $100M month for companies in the cell therapy space
categoriaRegenerative Medicine commentoComments Off on Another > $100M month for companies in the cell therapy space | dataMay 6th, 2012
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Predicting the Success of the Late-Stage Cell-Based Cancer Immunotherapy Pipeline?

By Dr. Matthew Watson

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Adam
Feuerstein
(phama and biotech writer with TheStreet.com) has designed his own rule. 

For those who know or follow Adam, this will come as no surprise.  He is
neither short of rules nor opinion and is never shy in his vivid expression of
either.  But this rule is more than a simple expression of informed
opinion. It was born of hard data analysis and has yet to be broken.  In
Adam’s own words, this is how he and his colleague (Mark J. Ratain) came to the
rule they coined the Feuerstein-Ratain Rule:
[We] analyzed the outcomes of
59 phase III clinical trials of cancer drugs going back 10 years, stratified by
the market value of the companies four months prior to trial results being
announced. What we found was a remarkable difference between the market values of companies that had positive and negative
announcements
.  (the list of companies/products used can be found here
)
Specifically, the median market capitalization was approximately
80-fold greater for the companies with positive trials vs. companies with
negative trials. There were no positive trials among the 21 micro-cap companies
(companies with less than $300 million market capitalization) whereas 21 of 27
studies reported by the larger companies analyzed (greater than $1 billion
capitalization) were positive.
The
editorial, entitled “Oncology Micro-Cap Stocks: Caveat Emptor!”, can be
found in Journal of the National Cancer
Institute
 
(JNCI) at http://jnci.oxfordjournals.org/content/early/2011/09/26/jnci.djr375.full.
They identified
drugs that were undergoing evaluation in phase III trials or for regulatory approval
by the US FDA between January 2000 and January 2009. 
They calculated the
company value based on the market value of primary drug sponsor roughly
three months prior to the release of the data.  They concluded that
whether or not a company had pharma in place was not determinative of a drug’s
success but rather that partnerships or acquisitions by Big Pharma can play a
role in determining a drug’s success only in that these deals may increase the
market value of the primary drug sponsor.  That value was the
determinative factor.
This
is Adam’s summary of the analysis they did that led to the “Feuerstein-Ratain Rule”.  
Below
are the important snippets from the analysis behind the rule:
The "Feuerstein-Ratain
rule" is derived from an analysis of 59 phase III clinical trials of
cancer drugs conducted over the past 10 years. We actually had no say
whatsoever in the selection of cancer drugs used in the analysis. The list was
put together by health economist Allan Detsky of Toronto's Mount Sinai Hospital
and his co-authors as part of their paper published in the Journal of the National Cancer
Institute
 suggesting that
doctors entrusted with conducting late-stage cancer drug clinical trials are
using advanced knowledge of the results of these pivotal studies to engage in
illegal insider trading.
Ratain and I used the same list of 59 cancer drug clinical trials,
re-analyzed by market value of the drug sponsors, to debunk Detsky's
insider-trading theory. That's how the "Feuerstein-Ratain rule" came
about, and we published our conclusions in the JNCI alongside Detsky's paper.
To restate our findings:  No positive trials among the
21 micro-cap companies
(companies with less than $300 million market
capitalization) whereas 21 of 27 studies reported by the larger companies analyzed
(greater than $1 billion capitalization) were positive
There were 21 companies on the
list with market values of $300 million or less, with a 0% success rate in
phase III cancer drug clinical trials.
The list also contained 11 companies with market caps between
$300 million and $1 billion. The clinical trial success rate for this mid-tier
or second strata group was 18%. (Two positive clinical trials out of 11.)
Lastly, there were 21 of 27 studies reported by the larger
companies analyzed (greater than $1 billion capitalization) that were positive,
or a 78% success rate.
So
what interesting for us in cell therapy?
It
is interesting to note that the Feuerstein-Ratain Rule is
limited to oncology drugs and all the companies behind them were public. 
Adam has not – nor has anyone else to the best of my knowledge – looked at how
the rule may or may not translate outside of oncology.

Of
the cell therapy companies to have received market approval in US or EU in the
past 10 years, one was public (DNDN) and one was still private (TIG) and went
public shortly therafter in the same year. TiGenix was a private company and is
not in oncology so the analysis arguably does not apply.  However,
Dendreon’s Provenge is an oncology ‘drug’.  Dendreon had a market cap of
about $430M in the 4 months before its ph III data was announced and as such
would have fallen in the 18% likelihood of success category.  That sounds
about right.

I thought it might be interesting to do our own look at what the Rule might say about the pipeline of late-stage cell-based oncology trials.  Following
is a list of cell therapy companies currently in ph III or II/III for oncology:

* Trial not expected to complete until Q1
2014 so a lot could happen to the market cap in 2012/13.  It also could be
argued that this is not an oncology treatment as per original data set but a
treatment of the side  effects of the primary cancer treatment.
** Trial not expected to complete until Q1
2014 so a lot could happen to the market cap in 2012/13.
*** It could be argued that this is not an
oncology treatment as per original data set but 
a treatment of the side effects of the primary cancer treatment.
+ It could be argued this is not a cell
therapy though we would argue it is.  Others might argue that as a phase II/III
trial with only 60 patients this may not be powered to be a pivotal oncology trial.
^ Trial currently in “suspension” so this
date may be pushed out or trial terminated. It also could be argued that this
is not an oncology treatment as per original data set but a treatment of
the effects of the primary cancer treatment.  Others might argue that as a phase II/III trial with
only 70 patients this may not be powered to be a pivotal oncology trial.
Conclusion:  At the moment it looks like both NovaRx and ERYtech will go to their phase III data completion (June and October 2012 respectively) as private companies.  To qualify under the rule, Cell Medica would have to go public within the year and/or Kiadis would have to go public within the next 25 months. 

The only
companies with cell-based oncology products currently in late-stage trials to
which the Rule would apply are Molmed’s HSV-TK and Newlink Genetics’ HyperAcute
Pancreas.  

Assuming both MolMed and NewLink's trials progress as planned, we won’t know what
they look like under the rule until around Sept 2013 at which time we can assess their
market cap against the Rule.  At the moment, it’s looking pretty bleak for
both of them according to the Rule though at least the NLNK price has been
going in the right direction of late.  

Certainly one would expect trading
volume to dramatically increase on both these as their trial completion dates
near.  It remains to be seen how this will impact price but they would
have to  dramatically increase in market cap (double or triple) to succeed
as the Rule predicts. 

Naturally, this is just one way of looking at the world and, of course, this rule - as with all rules - is meant to be broken.
  

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To Read More: Predicting the Success of the Late-Stage Cell-Based Cancer Immunotherapy Pipeline?
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2011 EMA Committee for Advanced Therapies (CAT) classification record. What can be learned?

By Dr. Matthew Watson

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What follows is the record of "classifications" done by the ATMP CAT in 2011 related to anything I would call "cell therapies". 


In my opinion there are a couple surprises. I'm surprised at the non-cardiac cells (MNCs, CD133s, and MSCs) for cardiac disease/repair being designated TEPs. I'm also surprised at the islets not being classified as an ATMP.


I've tapped into my European and/or regulatory colleagues to help explain those two as well as help us draw any other conclusions or observations we can make in terms of how the CAT is thinking based on the compendium of classifications we have to-date.  I'll post an update here when I have something useful.
______


In January, the following product was classified as a tissue engineered product - not combined:

  • Layer of autologous corneal epithelium containing stem cells intended for the treatment of extended corneal lesions



In April, the following product was classified as a tissue engineered product, combined: 

  • Allogeneic human fibroblasts cultured onto a biodegradable matrix, intended for use of conditions in the therapeutic area of dermatology



In May, the following product was classified as a somatic cell therapy medicinal product: 

  • Heterologous human adult liver-derived progenitor cells, intended for the treatment of inborn errors of liver metabolis



In July, the following product was classified as a Tissue Engineered Product, non-combined:

  • Suspension of allogeneic bone-marrow derived osteoblastic cells, intended for the treatment of non-union, delayed union or other fractures. 



In September, the following product was classified as a Tissue Engineered Product, non-combined:

  • Autologous mesenchymal stem cells (MSC), intended for the treatment of chronic heart failure symptoms by improvement in exercise capacity of NYHA class II and III chronic heart failure patients receiving standard therapy

     and the following product was not classified as an ATMP: 

  • Human islets of Langerhans, intended for: Post pancreatectomy for benign pancreatic pathologies (autologous); Treatment of severe forms of type 1 diabetes (Allogeneic)



In October, the following product was classified as a somatic cell therapy medicinal product: 

  • Autologous dendritic cell (DCs) immunotherapy consisting of autologous mature DCs coelectroporated with autologous RCC IVT RNA and synthetic CD40L IVT RNA, intended for the treatment of patients with advanced renal cell carcinoma



In November, the following products were classified as tissue-engineered products:

  • Concentrate of autologous bone marrow mononuclear cells (MNC), intended for improvement of heart function and quality of life in patients with chronic ischaemic heart disease and after MI.
  • CD 133+ Autologous bone marrow derived stem cells, intended for Improvement of heart function (LVEF) and quality of life in patients with chronic ischemic heart disease and after MI



In December, the following product was classified as somatic cell therapy medicinal product:

  • Autologous CD4+ T cells targeted to cells presenting class II restricted epitopes, intended forthe treatment of autoimmune diseases with MHC restricted specific immunity e.g. multiple sclerosis, type I diabetes or graft rejection.

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To Read More: 2011 EMA Committee for Advanced Therapies (CAT) classification record. What can be learned?
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Inactive and recently failed or terminated phase III or II/III cell therapy trials

By Dr. Matthew Watson



In the two previous posts I have outlined what I believe to be the active phase III and II/III cell therapy trials, as well as the cell therapy products to have 'recently' obtained formal regulatory market approval in some jurisdiction.


In the course of doing that work, I came across the following industry-sponsored phase III cell therapy trials which appear to be inactive and those which failed or were terminated.


INACTIVE INDUSTRY PHASE III    

  •     Aastrom              BRCs
  •     Aldagen              ALD-101
  •     Arblast               AMT-301
  •     Avax                  Mvax
  •     HepaLife Tech      HepaMate
  •     KeraCure             KeraPac
  •     t2cure                BMCs
  •     TVAX                 TV-Brain
  •     TVAX                 TV-Kidney-1

   
RECENTLY FAILED /TERMINATED INDUSTRY PHASE III   

  •     ABH (now Shire)        Dermgraft
  •     Cellerix                    Cx401

I don't imagine this is an exhaustive list but as I have encouraged in previous posts, I welcome feedback as to errors, corrections, or omissions.   I'm using the 2009-11 time-frame here.  I'll update the post accordingly.

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To Read More: Inactive and recently failed or terminated phase III or II/III cell therapy trials
categoriaRegenerative Medicine commentoComments Off on Inactive and recently failed or terminated phase III or II/III cell therapy trials | dataDecember 18th, 2011
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Recently approved cell therapy products

By Dr. Matthew Watson

Following is a list of cell therapy products approved recently (2010-11):    

  •  Dendreon                           Provenge                           US
  •  FCB-Pharmicell                 Hearticellgram-AMI           Korea
  •  Fibrocell Sciences              Laviv                                  US
  •  Living Cell Technologies    DIABECELL                     Russia

 Honorable mention goes to TiGenix' ChondroCelect approved in late 2009 representing the first EMA approval of an ATMP:

  • TiGenix                              ChondroCelect                   EU

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To Read More: Recently approved cell therapy products
categoriaRegenerative Medicine commentoComments Off on Recently approved cell therapy products | dataDecember 18th, 2011
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Active phase III or II/III cell therapy trials

By Dr. Matthew Watson

I know that the moment I publish anything that purports to be comprehensive there will be errors and omissions.  At the very least it will almost imminently be out-of-date in a fast-moving sector like cell therapy.
Nonetheless, because there is no other reliable repository of this information, I am daring to put this out there and hope you will feel obligated to assist me in ensuring its accuracy rather than critical of the effort.
What follows is what I currently believe to an current and relatively comprehensive list of phase III or II/III cell therapy trials around the world.  I am more confident of the industry list than the academic one.  More confident of its completeness and accuracy for North American and Europe than of Asia (particularly China). 
There is a spreadsheet behind this that includes more data fields like therapeutic category, cell/tissue source, cell type, expansion, indication, expected completion date, clinical trial site locations, etc.  If you want a copy of it, just email me (I'm not hard to find) or comment below.

INDUSTRY PHASE III or II/III (active or expected to be active in 1H 2012)  

       
    Aastrom                         Ixmyelocel-T
    Baxter                            ACT34-CMI
    Bioheart                         Myocell
    Cardio3 Biosciences         C-CURE
    Cardio3 Biosciences         C-CURE
    Cell Medica                     adoptive cellular therapy
    Cook Myosite                  AMDC
    Cytori                            ADRCs
    GamidaCell - Teva           StemEx
    Genzyme                        MACI
    Harvest Technologies       SmartPReP 2 BMAC
    Innovaell                        IES13 (Urocell?)
    Kiaidis Pharma                ATIR
    Miltenyi                         CliniMACS CD34 Selection System
    Medipost                       Cartistem
    MolMed                         TK
    Newlink Genetics            HyperAcute Pancreas
    NovaRx                         Lucanix
    Osiris                           Prochymal
    Osiris                           Prochymal
    Pervasis                        Vasugel
The 21 active or imminently active cell therapy industry-sponsored trials listed above break down as follows:
    • 52% (12) are autologous
    • 33% (7) are allogeneic
    • Two are gene-modified allogeneic
    • One involves autologous and allogeneic cells
    • 24% (5) are for cardiac-related indications
    • 33% (7) are for oncology or related indications
    • Two are for cartilage repair

 
* ACADEMIC PHASE III or II/III
  • Assistance Publique - Hôpitaux de Paris (France)
  • Association of Dutch Burn Centres (Netherlands)
  • Barts and The London NHS Trust (UK)
  • Erasmus Medical Center (Netherlands)
  • European Group for Blood and Marrow Transplantation (Europe)
  • Leiden University Medical Center (Netherlands)
  • Meshalkin Research Institute of Pathology of Circulation (Russia)
  • Meshalkin Research Institute of Pathology of Circulation (Russia)
  • Ministry of Health (Malaysia)
  • Royan Institute (Iran)
  • Rush University Medical Center, University of Sao Paulo, Uppsala University (US, Brazil, Sweden)
  • Third Military Medical University (Chia)
  • University of Minnesota, Masonic Cancer Center (US)
  • University of Minnesota, Masonic Cancer Center (US)
  • University Hospital of North Norway (Norway)
  • University of Utah (US)
* Active trials only - excludes trials which appear inactive, abandoned, and/or are stem cell transplant in oncology.  Primary source is CinicalTrials.gov.
___________________________________
I will try to keep this list updated at least once-per-quarter and indicate the date of the last update at the top of the post.
I eagerly encourage all readers to comment below or email direct with any errors and/or omissions.

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Sabrina Cohen Foundation Thanks Stem Cell Researchers

By Dr. Matthew Watson

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I'm proud to use every available resource at our disposal, including this blog, to highlight the efforts of the charity we support - especially during this holiday season.  


I would be so delighted to have you join me in supporting Sabrina Cohen and her efforts.  You can start by buying next year's calendar!



The Sabrina Cohen Foundation
The Sabrina Cohen Foundation for Stem Cell Research (SCF) is an internationally recognized nonprofit organization dedicated to building a global network of top scientists and clinicians in the field of Regenerative Medicine, while simultaneously funding cutting edge research and innovative therapies that will reverse spinal cord injury and effectively treat other impairments of the Central Nervous System.


The ‘CELLebrity’ Doctors Calendar
The 2012 CELLebrity Doctors calendar is now available for purchase from http://www.CELLebrityDocsCalendar.com.  All proceeds from calendar sales benefit the Sabrina Cohen Foundation for Stem Cell Research, a 501c3 non-profit organization directly funding stem cell clinical research.   



--Lee


_________________________________________________________________



On the night of November 12, David Porosoff’s Artrageous Gallery hotspot was converted into something likely never imagined -- a hotbed of stem cell research.  Sabrina Cohen fused the vividly artistic backdrop and venue with gambling, cuban music, great food, and beautiful people all to further her mission of raising money and awareness for stem cell research.

Dr. Sally Temple with Sabrina Cohen

Dubbed the Havana Casino Night, the event had several highlights including the granting of the 2011 Sabrina Cohen Foundation award to stem cell researcher Dr. Sally Temple.  


Representing the 3rd recipient of the annual SCF award, Dr. Sally Temple is studying how neural progenitor cells may be employed to create cell-based therapies for neurodegenerative disorders.  Dr. Temple is the co-Founder and Scientific Director of the Neural Stem Cell Institute located in Rensselaer, NY.  NSCI is the first independent, non-profit stem cell research institute in the USA.


The night, sponsored in part by DMR, Evensky & Katz and Harke Clasby & Bushman, raised $10,000 which will be dedicated toward next year’s SCF Award for Stem Cell Research.  


The event also marked the lauch of the the Foundation’s 2012 CELLebrity Doctors Calendar, this year featuring women in the field of stem cell research.  The calendar features academics, industry executives, physicians, and advocates primarily from the United States but also representing Sweden, Australia and Canadian covergirl, Dr. Fiona Costello.


“In science you don't have to accept anything anyone tells you, you can come up with a hypothesis and test it yourself. And you can be the first one to do it,” says Dr. Costello, whose research focus is on multiple sclerolsis and other impairments of the central nervous system. 


“Stem cell science is often accused of being ‘hyped,” says Cohen, “but that doesn’t necessarily translate into monetary support for or society recognition of the enormous contributions made by stem cell resarchers.  They often toil in anonymity making significant discoveries at great personal sacrifice.  I consider it my job to find a way to financially support their work and bring profile to them as people.”  


The ‘CELLebrity’ Doctors Calendar
The 2012 CELLebrity Doctors calendar is now available for purchase from http://www.CELLebrityDocsCalendar.com.  All proceeds from calendar sales benefit the Sabrina Cohen Foundation for Stem Cell Research, a 501c3 non-profit organization directly funding stem cell clinical research.   


The Sabrina Cohen Foundation
The Sabrina Cohen Foundation for Stem Cell Research (SCF) is an internationally recognized nonprofit organization dedicated to building a global network of top scientists and clinicians in the field of Regenerative Medicine, while simultaneously funding cutting edge research and innovative therapies that will reverse spinal cord injury and effectively treat other impairments of the Central Nervous System.


Sabrina Cohen is the Executive Director and President of the foundation. She graduated from the University of Miami with a degree in Communications, double majoring in Advertising and Psychology, and holds a post-graduate degree in Copywriting from the Miami Ad School.  She is a C5 Quadriplegic, as the result of a spinal cord injury from a car accident in 1992. In 2006, she established SCF to raise funds for research because she believes the field of Regenerative Medicine will lead to the greatest advances of our time. Sabrina is a Motivational Speaker & Spokesperson continuously speaking in schools, universities and community centers. She has spoken at scientific conferences around the country, including the "World Stem Cell Summit" at the University of Wisconsin, Harvard University, Stanford University, Baylor College of Medicine at the University of Texas, and at the United Nations. Sabrina believes her wheelchair is a vehicle to promote change.  


Sabrina Cohen was recognized by WebMD Magazine as a 2009 "American Health Hero”.  Sabrina is currently available for interviews highlighting the 2012 “CELLebrity” Doctors Calendar.



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Commercializing Cell-based Regenerative Medicines

By Dr. Matthew Watson

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When introducing a regenerative medicine cell based product to a commercial setting, there are a host of things to take into consideration to ensure a commercially viable and safe product for patient use.

In this QandA interview by Pharma IQ, William Fodor, Director of Translational Sciences, Cell Therapy Group, gives some teasers into a few of issues to keep in mind relative to commercial manufacturing scale?up of cell therapies.

Listen to the podcast here (registration required) or read the transcript below:

Pharma IQ:  Can you give some advice on the best way for a company to develop standards for
commercialization to improve safety?

William Fodor:  Well, with any biological product, you have to do all the appropriate testing and there’s really no standards necessarily to be developed by the company because the regulatory process is pretty well outlined by the FDA and the CBER Division and cell therapy products are regulated by the office of Cell Tissue and Gene Therapy Division.  So, it’s not that you need to develop standards for  commercialization to improve safety.  You need to follow the regulations involved by demonstrating to the FDA that your product is safe, and maintains the identity, in other words, your product doesn’t change during your regular manufacturing process.  Purity and then potency are all assays that need to be developed within the manufacturing process for your particular cellular product.


Pharma IQ:  And what are some approval processes and pitfalls to be aware of within the
scale?up process?

William Fodor:  So as you are scaling up, you absolutely need to maintain current good
manufacturing practices ? it’s known as cGMPs.  Typically, during a phase one, you can get
away with certain reagents that may not be fully GMPs.  Or in other words, if you use a growth
factor or a certain media that doesn’t have or isn’t manufactured under full GMPs,  as long as you test that particular reagent or media that you are using to ensure safety and sterility, you can typically get away with that in the phase one clinical trial process.  But when you move to a phase two, you need to make sure that all your reagents and medias and any compounds that come in contact with your product are all manufactured under good cGMP.

Pharma IQ:  What are some technology transfer and patent protection concerns to be
cognizant of?

William Fodor:  Well, with any cellular?based product, if there’s a technology that is out there
that a company wishes to pursue, to improve yield, or the manufacturing process, you need to
demonstrate that that technology fits within your manufacturing process.  So typically, what is
done is you’ll do validation runs to ensure that that new technology satisfies the regulatory
process for your manufactured product. With respect to patent protection, again, that company needs to maintain their IP portfolio and needs to make sure that they’re not infringing other intellectual property and that’s just standard for the industry.

Pharma IQ:  And do you have any tips for ensuring quality and consistency no matter how little
or how much one is producing?

William Fodor:  Yes, when you manufacture a cell?based product, it’s not that much different than any other biologic product.  And so, whenever you do manufacture, whatever scale it is, you have to ensure safety, and that’s sterility, tests for microplasma, or other adventitious agents; things like bioburden and endotoxins, so all those tests need to be performed. You need to have an identity test to make sure that your cell product ,whatever scale your manufacturing is, that at the end of that manufacturing run, the product hasn’t changed.   Again, no matter what scale you’re at, you need to make sure the identity of the product is
consistent from batch to batch.

For identity, you can do a number of things, and again, for a cell?based product, if you want to look at cell surface antigens to ensure that the cell surface proteins on your cellular product don’t change over time or through your manufacturing process.  And typically, what you like to do is keep it relatively simple.  You don’t want to test for a hundred things because you’re just asking for the potential for something within those hundred things to change.  So typically, what you do is maybe three to four cell surface antigens to ensure your product identity is consistent and you can also do PCR to determine that an intracellular protein of interest doesn’t change during your manufacturing process.

You also need to ensure for purity, so you want to quantitate your active cell or your tissue type.  And then potency; you need to demonstrate the product has a consistent potency and the biological activity of that final product doesn’t change during the manufacturing process.   And then typically, what you do is you archive.  You archive samples from during your manufacturing process. You cryopreserve those so you can always go back to ensure that that a particular batch was consistent with other batches that were manufactured.
...

Join Dr. Fodor and other industry leaders in Philadelphia, December 12th and 13th 2011 for the IQPC Commercialization of Regenerative Medicine Summit.  For more information or to register, visit
http://www.regenerativemedicinesummit.com.

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To Read More: Commercializing Cell-based Regenerative Medicines
categoriaRegenerative Medicine commentoComments Off on Commercializing Cell-based Regenerative Medicines | dataNovember 13th, 2011
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Cell Therapy & Regenerative Medicine Domains Available

By Dr. Matthew Watson

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Please pardon the crass commercial nature of this post.  I try to keep self-promotion to a minimum here but I'm looking to unload a number of domains I have the related to cell therapy and regenerative medicine and thought this might be the easiest way to get the word out.  Let me know if you are interested in any of the following: 


anticyte.com*
biocellutions.com***
.
cardiacregenerativemedicine.com* 
cardiacregenmed.com* 
cardiactissuerepair.com* 
.
cellexpertsolutions.com**
.
celltherapyalliance.com* 
celltherapyassays.com*** 
celltherapycellutions.com**** 
celltherapycompany.com**** 
celltherapyconsortium.com* 
celltherapydevelopment.com***
celltherapymanufacturing.com**** 
celltherapymarketing.com* 
celltherapypartner.com* 
celltherapypatents.com**** 
celltherapysolution.com*** 
celltherapystrategies.com*** 
celltherapysupport.com* 
celltheraytech.com** 
celltherapytechnologies.com*** 
celltherapyventures.com*** 
celltherapyworks.com***
.
cellutionstrategies.com*** 
.
commerciaizingcelltherapy.com** 
commercialisingcelltherapy.com* 
commercializingcelltherapies.com** 
commercialisingcelltherapies.com* 
.
cordbloodshipper.com*** 
cordbloodshipping.com*** 
.
developingcelltherapies.com* 
.
diagnostacell.com* 
diagnostacells.com* 
diagnosticstemcells.com** 
.
discoverystemcells.com** 
.
expertcellutions.com***
.
integratedcelltherapycellutions.com** 
integratedcelltherapysolutions.com**
.
ipsbiologics.com** 
ipsbiomedical.com**
ipsbioscience.com**
ipsbiosystems.com**
ipsbiotechnologies.com** 
ipslifesciences.com**
.
longtailbiomedical.com***
longtailtherapeutics.com*** 
longtailtherapies.com*** 
.
personalizedcelltherapy.com*** 
.
regenerativecellutions.com*** 
regenerativemedicinepatents.com****
.
stemostics.com** 
stemomics.com** 
stemonics.com*** 
.
stemcellcollect.com*** 
.
strategiccellutions.com** 
strategicell.com*** 
.
thecelltherapycompany.com****
.
totalcelltherapysolution.com***
totalcelltherapysolutions.com*** 


Funky ones: 
 cellenterprise.com* 
 cellsforce.com* 
 cellsinnovations.com* 
 cellsmarket.com* 
 cellspitch.com*
 cellsrep.com* 
 cellstech.com* 
 cellsventure.com*
 iregener8.com** 


I have others - some of which are variations of ones listed above (such as with "the" in front) and would be available - some of which I'm still wanting to hold.
 _____________________________ 


 * ~$1,500 
** ~$5,000 
*** ~$10,000
**** ~$20,000

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To Read More: Cell Therapy & Regenerative Medicine Domains Available
categoriaRegenerative Medicine commentoComments Off on Cell Therapy & Regenerative Medicine Domains Available | dataSeptember 25th, 2011
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Commercial-stage Cell Therapy Companies and Products

By Dr. Matthew Watson

Below is sample list of companies with cell therapy products* on the market in Europe, USA, or Japan.

Company                                                    Product              
Advanced BioHealing (now part of Shire)       Dermagraft
Aliktra                                                       MySkin
Avita Medical                                             ReCell® Spray-On Skin
Bio-Tissue                                                 Prokera
Bio-Tissue                                                 AmioGraft
BioTissue Technologies                              BioSeed-C
BioTissue Technologies                              chondrotissue
Cytori                                                        Celution System
euroderm                                                   Epidex
euroderm                                                   EpiGraft
Fidia Farmaceuitici                                     Hyalograft 3D
Fidia Farmaceuitici                                     Laserskin
Fidia Farmaceuitici                                     Hyalograft C
J-TEC Epidermis                                       Japan Tissue Engineering Co.
J-TEC Cartilage                                         Japan Tissue Engineering Co.
J-TEC Corneal Epithelium                          Japan Tissue Engineering Co.
Nuvasive                                                    Osteocel Plus
Provenge                                                    Dendreon
Sanofi (previously Genzyme)                       Epicel
Sanofi (previously Genzyme)                       Carticel
TiGenix                                                      ChrondroCelect
Therakos                                                    Therakos Photopheresis

* This list does not purport to be exhaustive of all cell therapy products legally sold in these regions.  This list  does not include approved products in other highly-regulated jurisdictions, such as Australia, New Zealand, or Korea, for example.  This list also excludes those cell-based treatments provided as a hospital or clinic-based service such as stem cell transplantation (hospital) or Regenexx (Regeneration Sciences, Inc.).

For the purposes of this list, “cell therapy” is defined loosely as any product which has in it live cells when administered to the patient including tissue transplants and devices.

Note that some of these products may be subject to emerging regulatory restrictions under the EMA ATMP regulations which may result in them having to be pulled from the market by the end 2012 at the latest.

If you would like to suggest any revisions or additions to this list, please do so in the comment section below.

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To Read More: Commercial-stage Cell Therapy Companies and Products
categoriaRegenerative Medicine commentoComments Off on Commercial-stage Cell Therapy Companies and Products | dataSeptember 18th, 2011
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Potential far-reaching implications of the ongoing fight over point-of-care autologous cell therapy

By Dr. Matthew Watson

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The FDA recently issued an untitled letter to Parcell Laboratories and its contract manufacturing organization, New England Cryogenic Center (NECC), pertaining to the product PureGen™ Osteoprogenitor Cell Allograft intended for the "repair, replacement, reconstruction of musculoskeletal defects". The letter stated:

"The PureGen™ Osteoprogenitor Cell Allograft is not the subject of an approved biologics license application (BLA) nor is there an IND in effect. Based on this information, we have determined that your actions have violated the Act and the PHS Act."

This would appear to be an indicator that the FDA does not intend to relax enforcement of its view of how autologous cell therapies are to be regulated despite its ongoing litigation on this very subject with RSI.

As followers of this blog know, since the battle's inception in 2008 I have followed the case of Regenerative Sciences, Inc and their war with the FDA over their right to provide certain autologous cell therapy treatments to patients in certain circumstances without FDA approval. My first blog entry on the topic was in September 2008 in which I pointed out that the FDA had written a letter to RSI that July taking issue with some of their practices.

My next blog on the subject was February 2009 in which I concluded "I think the FDA is building its case and a showdown is on its way to Denver-town."

At the advice of legal counsel, I pointed out in a March 2010 blog entry that my reference to FDA's July 2008 letter to RSI was not officially a "warning letter" as that is defined and as I had referred to it but rather an "untitled letter". I also commented that FDA's lack of enforcement action against RSI to-date was emboldening medical practitioners into thinking FDA was reconsidering their position on the legality of providing autologous, expanded cells to patients outside of an FDA-cleared IND or BLA.

I took some satisfaction in announcing on my blog in August 2010 that the FDA had finally taken action against RSI. My satisfaction was not rooted in a belief that the FDA is right (I've always been agnostic as to which side is right) but in the sense that things had occurred as I had predicted they would.

The action the FDA chose to take against RSI was to seek an injunction against RSI from continuing to provide the "offending" treatment - a version of the Regenexx™ procedure using mesenchymal stem cells (“MSCs”) grown outside the body after harvest for the later infusion back into the donor-patient for the treatment of various orthopedic conditions - which the FDA alleges is a "product" falling under its regulatory authority but for which RSI has never received any FDA clearance to provide to patients.

RSI counterclaimed against the United States, challenging the FDA’s authority to regulate the Regenexx™ Procedure in question and challenging certain FDA regulations. The United States moved to dismiss Defendants’counterclaims and for summary judgment.

I have continued to follow the case relatively closely through a number of source (see this sample media coverage in OthosSpineNews) as it continued to progress. Last month, for example, a blog I follow posted an eloquent case in support of RSI's position with the help of Mary Ann Chirba, J.D., D.Sc., M.P.H. of Boston College Law School.

So it was with much interest that I was recently notified of an order issued by the court which appears to have the potential to take the case in a very unexpected direction with enormous potential ramifications.

The context is that the judge was reviewing the FDA's motion for summary judgment, RSI's response, and FDA's reply when the judge issued this order to show cause.

At its essence the Judge has ordered the FDA to file a brief no later than 26 September showing the court why the term “chemical action” applies to stem cells. It is is a short Order the core of which reads as follows:

The Government finds its definition for a “drug” in the FDCA: “The term ‘drug’means . . . articles intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease in man or other animals” and “articles (other than food) intended to affect the structure or any function of the body of man or other animals.” 21 U.S.C. § 321(g)(1)(B)&(C).

This definition, at least as to subsection (C), would be broad enough to encompass a boot on a patient’s ankle to hold it secure after ankle surgery. The Court doubts that was Congress’s intent.

Neither party references the definition for “device,” found in the statute at 21 U.S.C. § 321(h). A “device,” is a certain kind of “article” used in diagnosis, cure, mitigation, treatment or prevention of disease, 21 U.S.C. § 321(h)(2), but which, presumably unlike a drug, “does not achieve its primary intended purposes through chemical action within or on the body of man or other animals and which is not dependent upon being metabolized for the achievement of its primary intended purposes.” 21 U.S.C. § 321(h).

These contrasting definitions immediately raise the question of why the Court should not interpret the meaning of the word “drug” to include not only an article for use in diagnosis, etc., and intended to affect the structure or function of a patient, but also an article that “achieve[s] its primary intended purposes through chemical action” and which is “dependent upon being metabolized for the achievement of its primary intended purposes.” Id.

The United States is ORDERED TO SHOW CAUSE why the Court should not read the definition of “device” at 21 U.S.C. § 321(h) as informing and restricting the definition of “drug” at 21 U.S.C. § 321(g)(1)(B)&(C).

It will be most curious to see how the FDA argues out of the corner that many believe the Judge has painted the agency. The FDA recently defined “chemical action” in its draft "Guidance for Industry and FDA Staff: Interpretation of the Term “Chemical Action” in the Definition of Device under Section 201(h)of the Federal Food, Drug, and Cosmetic Act". What is curiously absent from the document is any mention of cells or HCT/P’s despite CBER’s approval stamp on the document.

Another line of argument centers around whether cells - notably 'stem' cells - are “metabolized" as that term is defind.

If one extrapolates the ramifications of where the court appears to be currently leaning, the implications of this judgment may have far-reaching implications for biologics in general well beyond cell therapy and certainly well beyond autologous cell therapy.

As some quite logically argue, one potential scenario is that this judge rules all biologics fail to fall within the legislative "drug" definition. The argument goes like this. The drug regulations live under title 21, which has narrow definitions for what constitutes a drug. The FDA’s authority over biologics comes from title 42, which is merely to control communicable disease transmission in transplants, with no authority to take the drug provisions from title 21 and apply them to title 42. So the agency is risking a loss of control over all biologics.

What’s curious here - and perhaps somewhat ironic for RSI at this stage - is that they only ever set out to challenge their ability to regulate autologous cells used by a physician as part of his or her medical practice yet now the FDA's authority to govern all biologics is currently under question.

The fact that I cannot fathom the courts striking FDA's jurisdiction over all biologics when the dust settles on this case does not make the arguments any less compelling and it does leave open the possibility that a lower court Judge such as the one presiding over this case may be inclined to make a ruling which essentially ensures the issues are punted to the appellate courts for a more considered ruling. In such circumstances, even if the FDA were to prevail at the end of the day (perhaps a decade down road) the uncertainty such a ruling would rain down on the sector would be commercially stifling - even if if were just limited to autologous cell therapy let alone if were any broader.

My dated and unpolished law degree can only take this analysis so far and anyone interested in some further but delightfully light and practical reading on the potential ramifications of the case could do no better than read a paper published recently by the relevant practice groups at the law firm K&L Gates entitled "Cultured Stem Cells for Autologous Use:Practice of Medicine or FDA Regulated Drug and Biological Product in which they review the case and its potential implications - the latter of which the authors are not guilty of underestimating in the following concluding sentence of their analysis:

The court’s decision will, to a large degree, dictate the types of legal strategies and business models that will be necessary to successfully perform stem cell procedures in the future.

_____________

post-script: The potential stink of commercial uncertainty wafting from this case is even more egregious when combined with the uncertainty around what to expect from the FDA in its much-anticipated and typically overdue guidance on adipose-derived cell therapies.

Rumor has it that the FDA is leaning toward considering most (if not all) means of deriving cell populations from adipose tissue (typically lipoaspirate) to be governed as what we colloquially refer to as a '351' thus taking it out of the purview of the practicing physician and into the hands of companies prepared to follow the traditional "drug development' model for new medicines. The rationale here is that the mechanical and/or enzymatic digestion required to separate the desired cell populations from the stroma take the process beyond "minimal manipulation'.

Watch for this guidance from CBER OCTGT in the weeks to come and/or any relevant rulings by the Tissue Reference Group. This would be a serious blow to those building business models around point-of-care, autologous adipose-derived cell therapy treatments.

What makes this even more interesting is the pace of which US-based medical practitioners (and/or companies supporting them) are adopting and selling autologous cell-based products, services and/or treatments for sundry indications in ways which many would argue are apparently in obvious and flagrant disregard for the FDA's regulatory authority over such treatments. Included for consideration on such a list would be the following:

IntelliCell Biosceinces

eternaMD

Arizona Stem Cell Center

.

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To Read More: Potential far-reaching implications of the ongoing fight over point-of-care autologous cell therapy
categoriaRegenerative Medicine commentoComments Off on Potential far-reaching implications of the ongoing fight over point-of-care autologous cell therapy | dataSeptember 18th, 2011
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Good Data? $100. Good Product Development? $100. Good Commercialization Strategy? Priceless.

By Dr. Matthew Watson

I'm not going to fool anyone into believing I'm a therapeutic product development expert but that's not going to stop me from making a few humble observations in light of the Dendreon "fiasco" of last week which I have no doubt will one day be considered an unfortunate pothole on their road to eventual success.

(though perhaps not before certain current management finds themselves polishing their CVs or retiring to spend their time alternating between their yachts and the courtroom defending their questionable stock trading antics)

I received the following message this morning by email. I don't include it here to promote or endorse or even comment on NWBT, DCVax, Linda Powers, or Toucan Capital in any way -- or in a way that is blind to all the things right or wrong about any of them -- but simply to illustrate the 3 points I want to make below the email.
NWBT HIGHLIGHTS COST EFFECTIVENESS OF DCVAX® IN VIEW OF RECENT IMMUNOTHERAPY PRICING CONCERNS

Northwest Biotherapeutics' (OTC.BB: NWBO)... DCVax® immune therapies for a broad range of cancers (including prostate, brain, ovarian and others) hold the promise, based on available data to date, of being cost effective and priced below other immune therapies while still providing substantial profit margins for the Company and longer survival for patients.

The investor concerns in the news relate to the pricing and reimbursement of Provenge for late stage, metastatic prostate cancer. Provenge is priced at $93,000 for one month of treatment and was approved by the FDA based upon having added 4.5 months of patient survival (to reach overall survival of 25.9 months).

NWBT’s DCVax® will be priced in the range of $37,000 per year for up to 3 years of treatments. In NWBT’s Phase I/II multi-center clinical trial in late stage, metastatic prostate cancer, DCVax® added 18 months of patient survival (to reach overall survival of 38.7 months). DCVax® has previously been cleared by the FDA for a 612-patient, randomized, controlled Phase III trial, although the trial has not yet begun. As is typical before a Phase III trial, the manufacturing processes and product costs have already been determined.
...
...

The key to the substantial pricing advantage of DCVax® is NWBT’s proprietary batch manufacturing process together with its cryopreservation technology for frozen storage of the finished vaccine. NWBT has spent a decade developing and improving its manufacturing and cryopreservation processes. The manufacturing of personalized, living cell products is expensive. But the frozen storage of living cells is quite low-cost – once the specialized freezing technology is worked out for a particular type of cells (the culture conditions, rate of freezing, density of cells and many other factors).

NWBT’s manufacturing methods produce – in a single manufacturing run – a large batch of personalized DCVax® product for 3 years of treatments are much less costly than separate manufacturing runs for each treatment. The technology for freezing the master immune cells (dendritic cells) which comprise DCVax® enables these
cells to remain frozen for years and, when needed, to be thawed and “come back to life” with full potency.

This approach makes DCVax® an "off the shelf” product [for that patient] for several years of treatments after just one manufacturing run. In contrast, Dendreon must do a separate manufacturing run for each one month of treatments. In addition, Dendreon's Provenge product is fresh and not cryopreserved, which limits its shelf life to at most a few weeks.

Another important factor in the cost effectiveness of DCVax® is its simplicity and ease of administration. DCVax® is delivered as a small intra-dermal injection under the skin, similar to a flu shot. As such, it can be administered in any physician’s office or clinic. There is no lengthy intravenous infusion, with the attendant patient discomfort, cost and need for a specialty infusion center. In contrast, Dendreon’s Provenge is delivered by intravenous infusion.

The cost effectiveness of NWBT’s DCVax® is enhanced by the fact that DCVax® is targeting a portion of the prostate cancer market that is 4 times the size of the market segment that Dendreon’s Provenge is currently targeting....

Now this is NWBT clearly blowing their horn - nothing wrong with that - in an attempt to woo back frightened investors. I'm agnostic as to whether any of it is true but it does serve to draw out several points of distinction between what some companies might do to optimize their products for commercial success versus what others might do in an overriding belief that clinical benefit is the only precursor to the happiness of investors, the physician community, patients, and partners.
What I say below is not a commentary, in criticism or praise, on any particular company including NWBT or Dendreon. Many others - Luke Timmerman among the best of them - have provided outstanding and in-depth analysis of the Dendreon story along the way.
I'm only interested in what we as an industry might learn from recent experiences or trends and to perhaps facilitate a useful discussion based on 3 simple observations from someone who has swum in this cell therapy pond for now just over a decade:
1. There is a tendency among some to believe that what we are making (cell-based therapies) are so revolutionary and compelling that the products should almost sell themselves - to investors, partners, regulators, insurers, physicians, and patients.
Of course we know it's not true but sometime we act like it is.
Appligraf and Dermagraft didn't sell themselves to physicians even after regulatory approval. Neither is Provenge apparently-- though they did a good job of selling it to very vocal patient groups. Organogenesis and Advanced Biohealing had to work very long and hard to get profitable reimbursement and market penetration. Dendreon will too.
Investing early in understanding potential clinical adoption hurdles, reimbursement issues, and how the product is and will be perceived not by its champions but by its critics and most importantly, the average agnostic practitioner, is not easy to do because it means spending precious resources long before there is a product to sell but it may mean the difference between a product which eventually sells and one which doesn't.
2. There is a tendency to de-emphasize what I call the "ancillary sciences" around a product -- like lowering the cost of goods, optimizing fresh or frozen storage, cell delivery (e.g., injection/application science) mechanisms, in vivo cell tracking, onsite clinical handling, etc.
Product development science is a science. The science that turns good data into something commercially viable. Not everyone is good at it and certainly this is where a lot of companies fail. PD is not the 'second cousin' in the room of esteemed basic and clinical science.
Take these examples when considering the cellular immunotherapy sector:
Several prominent immunotherapy investigators I have spoken with strongly believe cell-based immunotherapies will require long-term administration to be meaningfully effective -- certainly longer than 3 doses in 3 months. Did Dendreon lock into their clinical protocol too early?
Other immunotherapy companies - like Opexa Therapeutics for instance - create multiple doses for a patient from a single patient collection thus saving considerable expense and creating a better patient experience. Did Dendreon lock into their manufacturing protocol too early?
Other companies are investing heavily in finding ways to extend viable shelf-life of their fresh products or to create cryopreserved versions of their product to optimize its commercial viability.
Imagine a Dendreon that only had to build one manufacturing facility (with a backup CMO) to serve the US market rather than 3 facilities. With a cryopreserved version of the product or a version that had longer than its current limited shelf-life (~72 hours I believe?) that might have been possible.
At every point in clinical development there must be concurrent R&D towards the product's:
  1. science (e.g., MOA, characterization, etc),
  2. clinical effect, and
  3. how to optimize its commercial viability - a big part of which is what we think of as 'product development'.
This is the 3-legged footstool of a commercialization strategy geared for success (credit to Bob Preti of Progenitor Cell Therapy for this analogy).
But 'product development' is also not always about the product strictly speaking and even an expansive definition of product development is only part of a good commercialization strategy.
There is significant component of it that is about studying ways to lower the cost of goods, improving manufacturability and scalability, how hard/easy it is to handle, the patient treatment experience, the physician experience, how it impacts patient's QOL (quality of life), not just what side effects it generates but what side effects it prevents (resulting from other treatments or no treatment) and the cost-savings that generates, etc.
Other cell therapy companies have been more proactive in terms of engaging not only KOLs but average practitioners in a meaningful way that might impact their product and clinical design as well as reimbursement and clinical delivery issues.
3. There is an understandable, largely VC-driven, desire to race forward to the next trial phase when a phase-repeat to optimize or better understand different aspects of the product might be the better way to go.

What's worse? Facing the prospect of not being able to get funding or a partner on the terms one wants for a 2nd phase II or burning through a bunch more money in phase III in an attempt to bring a product to market that isn't market ready? I understand its a tough choice -I'm not saying it's an easy one - but one is certainly more strategic and, as pharma says, is certainly a "de-risking" pathway.
Consider the ratio of products we've seen thus far in the cell therapy industry's short life-span that have been raced to phase III or (worse yet) market only to seriously stumble if not fail when they get there. I can think of 8-10 off the top of my head and there are less than 20 cell or tissue based therapies on the market in US/EU that have received any kind of formal regulatory approval.
Some would argue that this is a prime example of why spinning companies out of academia too early is not beneficial because companies want to minimize exploratory science and lock into a "product" too early. I would argue that this may be true if the problem is understanding the product characterization or mechanism of action but not if your problem is related to how best to develop/optimize the product for commercial viability. Few academics are geared to think this way.
Summary
I certainly don't believe Dendreon failed to identify or consider each and every one of the things they might have done better along the way. I'm sure they did and sure they made calculated judgement-calls about how to approach each one.
Since I'm not a shareholder I don't have to worry myself about being critical of their decisions but rather simply to do what I can to ensure that we as an industry do our best to learn from what - with the benefit of retrospect - may be apparent they did right and wrong.
What are your thoughts? To what extent are the problems that Dendreon has experienced along the way with PROVENGE a predicable result of it being a first-generation product or the result of insufficient focus on critical investigation into the less sexy "ancillary sciences" of product and commercial optimization?
(comment below and/or in the LinkedIn Cell Therapy Industry Group)



To Read More: Good Data? $100. Good Product Development? $100. Good Commercialization Strategy? Priceless.
categoriaRegenerative Medicine commentoComments Off on Good Data? $100. Good Product Development? $100. Good Commercialization Strategy? Priceless. | dataAugust 14th, 2011
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Clinical trial costs

By Dr. Matthew Watson

Based on a survey* of 21 drug makers, 12 biotechs, nine device makers and 23 contract research organizations, PharmaLot has recently published the following metrics for current clinical trial costs (see full article here)
Average per-patient trial costs across all therapeutic areas:
Phase I: $21,883
Phase II: $36,070
Phase IIIa: $47,523
Phase IIIb: $47,095
Phase IV: $17,042
Average cost per patient for a cardiovascular trial:
Phase II: $33,700
Phase IIIa: $21,750
Phase IIIb: $6,830
In oncology, the average per patient trial cost:
Phase II: $73,303
Phase IIIa: $57,207
Phase IIIb: $65,900
For central nervous system disorders:
Phase II: $28,197
Phase IIIa: $33,768
Phase IIIb: $41,824
For diabetes:
Phase II: $ 8,854
Phase IIIa: $12,667
Phase IIIb: $10,700
Anyone have any thoughts or data as to how this compares to cell therapy trials?
--Lee
*survey conducted by Cutting Edge Information.

To Read More: Clinical trial costs
categoriaRegenerative Medicine commentoComments Off on Clinical trial costs | dataJuly 31st, 2011
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Cell Therapy’s Got Talent Technology Showcase – A Call for Cell Therapy Manufacturing Technology Presentations

By Dr. Matthew Watson


in collaboration with:

In an effort to showcase the latest technologies driving the production of cell therapies, the Cell Therapy Group and Informa Life Sciences are proud to announce the introduction of the "Technology Showcase" session and award to be held in conjunction with Informa's Cell Therapy Manufacturing conference to be held 30 November to 1 December 2011 in Brussels Belgium.

Having held the same conference last year in London, Informa is committed to building on the success of last year's event by continuing to create a meaningful European forum for the issues related to the clinical and particularly commercial-scale production of cell-based therapies.

The Technology Showcase session, taking place on the main agenda, will feature 6 x 10 minute presentations from innovative companies developing cutting-edge technologies in the field of cell therapy manufacturing, and is particularly relevant to SME and academic groups with limited marketing resources.

All presentations will be reviewed by the Scientific Advisory Board with the winner announced at the end of the session. Exposure on BioProcess International's website is also included.

Technologies we'd like to promote include:

  • Manufacturing systems including bioreactor technologies
  • Cell harvest/collection technologies
  • Cell storage/logistics technologies
  • Clinical cell delivery and/or other point-of-care technologies
  • Automation technologies
  • Cell separation system
  • Cell process devices
  • Innovative reagents, scaffolds, matrices, and other “ancillary” tools
  • Technologies to close currently open systems
  • Suspension-based production systems
  • Disposable technologies

How to apply:

To apply to present companies must submit an abstract (<300 words) to daniel.barry@informa.com and lbuckler@celltherapygroup.com outlining the product or service to be presented and why it is a critical technology related to cell therapy manufacturing.

The deadline for applications is SEPTEMBER 15 2011 - Priority given to early submissions

    The cost of taking part in the Technology Showcase is £2,700 which includes the following benefits:

    • 1 x 2-day conference pass (normal price £1,599)
    • 10-minute podium presentation within main conference room
    • 1 poster display in the Exhibition Hall
    • Marketing - company logo displayed on website and event guide
    • Exposure in BPI Magazine

    Terms and conditions:

    To be eligible the product or service to be presented must be:

    • On the market for no less than 2 years or expected to be on the market no later than Q4 2012
    • Appropriate for, applicable to, and compliant with clinical-grade manufacturing requirements (technologies only available for research use will not be considered)

    Plus...

    • The company must have no more than 15 employees
    • The company has been running for no more than 5 years, and
    • The company generates annual revenue of no more than $5m

    For further information please contact: daniel.barry@informa.com or lbuckler@celltherapygroup.com

    To Read More: Cell Therapy’s Got Talent Technology Showcase – A Call for Cell Therapy Manufacturing Technology Presentations
    categoriaRegenerative Medicine commentoComments Off on Cell Therapy’s Got Talent Technology Showcase – A Call for Cell Therapy Manufacturing Technology Presentations | dataJuly 10th, 2011
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    In vivo cell trafficking just took a leap forward

    By Dr. Matthew Watson


    Today Celsense, Inc. and the University of Pittsburgh Cancer Center announced that the FDA has authorized the use of the Cell Sense imaging reagent for use in a phase I clinical trial of a dendritic cell caccine to treat colorectal cancer patients.

    This is the first FDA authorization of the use of Cell Sense in patients. Cell Sense is a novel perfluorocarbon tracer agent used to safely and efficiently label cells ex vivo without the use of transfection agents. Labeled cells are then transplanted into the patient enabling researchers and clinicians to non-invasively track the administration and migration of therapeutic cells using MRI. Applications include tracking cells in immunotherapy or regenerative medicine as well as the diagnosis of inflammatory sites by tracking selected populations of immune cells.

    Cell Sense has been studied extensively in preclinical testing with many different human cell types including human cells in animals. For instance, in 2009 a paper was published in Informa's Cytotherapy, in which Celsense’s novel perfluorocarbon tracer agent (product “Cell Sense”) was used to label human DCs ex vivo for the purpose of tracking the cells in vivo post-transplant by 19F MRI. The paper provided an assessment of the technology and demonstrated that human DCs were effectively labeled without significant impact on cell viability, phenotype or function. Furthermore, the labeled dendritic cells were clearly detected in vivo by 19F MRI in a model system, with the labeled cells being shown to migrate selectively towards draining lymph node regions within 18 hours after transplant.

    Many investigators looking at various ways to label cells to enable in vivo imaging have expressed concern that the FDA would delay the regulatory progress of their therapeutic candidates if an imaging modality was introduced.

    This concern is based on numerous reports of MRI contrast reagents, such as the commonly investigated USPIO (ultrasmall superparamagnet iron oxide), deleteriously affecting the cells (see recent paper in Cell Transplantation).

    "We believe that the authorization of this IND will alleviate such concerns and lower the barriers for adoption. The agency’s tangible support for bringing new technologies to bear in the translation of cell-based therapeutics is very encouraging,” s Charlie O'Hanlon, President and CEO of Celsense.

    While there have been approved uses of imaging reagents (e.g., Feridex, etc) with cell therapies in other countries (e.g. Isreal), I believe this may be the first FDA-sanctioned use of a particle-based imaging label with a cell-based therapy. Other approaches to cellular imaging include nuclear imaging reagents and genetically modifying cells with reporter genes such as those provided by CellSight Technologies.

    Imaging labels are capable of providing investigators with data demonstrating where the cells go, at what volumes, and for how long they stay at the target location.

    The industry has been keen to see these kinds of technologies clinically employed but different cell-based labels have created their own technical, clinical, and/or regulatory hurdles. I'm hopeful that Celsense and others like them are now ushering us into a new era where we will eventually be able to use various technologies to monitor and collect valuable data concerning cells after they have been administered as a therapy to a patients.

    Additional resources on the topic of imaging for cell therapies:

    CIRM recently hosted a webinar - "CIRM/RMC Webinar: Imaging Technology for Cellular Therapies. One of the speakers, Dr. Shahriar Yaghoubi from CellSight Technologies, provides an overview of cell therapy imaging with emphasis on PET. Click hear for the archived playback.

    A very interesting article posted today on Harvard's StemBook website. "In-vivo Stem Cell Imaging - Regulatory Challenges and Advances". Nice overview intel from J. Bulte and a snapshot into E. Wirth's (of Geron) perspective re: stem cell imaging.

    A new book from CRC Press edited by Dara Kraitchman and Joe Wu will be out soon. It gathers together different methods for comparison. The issue will remain the sensitivity of the methods to track few cells. "Stem Cell Labeling for Delivery and Tracking Using Non-Invasive Imaging".

    MRI contrast agents can change stem cell proliferation

    There s also a very informative discussion thread on the topic in the Cell Therapy Industry group on LinkedIn.

    To Read More: In vivo cell trafficking just took a leap forward
    categoriaRegenerative Medicine commentoComments Off on In vivo cell trafficking just took a leap forward | dataJuly 3rd, 2011
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    Cell Therapies: Commercializing a New Class of Biopharmaceuticals

    By Dr. Matthew Watson


    Over the past six months I have been honored and pleased to have seen and been part of an increasing focus and attention being paid to the unique manufacturing and bioproduction issues related to cell therapy.


    Certainly it is the Cell Therapy Group's view, that this is both timely and much-needed as more cell therapies reach later-stage. Issues related to larger scale production and lowering the costs associated with it will be critical to successful commercialization of these products. It is encouraging to see both content-providers and and companies involved in potentially bringing solutions to these issues now bringing their focused energies to this sector.

    This focus has come from a number of different sources including conferences focused solely on the topic, companies engaging stakeholders in identifying potential bottlenecks they might be positioned to solve, more conference sessions dedicated to these issues, and now a commitment by one of the leading publications in bioprocessing to engage both the cell therapy industry and the traditional bioprocessing community in stepping up the level of two-way education, dialog, and problem-solving that will be critical to commercializing these products.

    In March/April 2011, watch for a special issue of BioProcess International entitled "Cell Therapies: Commercializing a New Class of Biopharmaceuticals".

    BioProcess International is a publishing leader of cutting edge technologies, improved processes and breakthrough sciences. With this cell therapy focused issue, in partnership with ISCT and others, BPI is launching what we hope will be a regular supplement and increased focus on the unique bioproduction issues related to cell-based therapies.

    BioProcess International aims to accomplish three main objectives with this supplement:
    • Educate the bioprocess and cell therapy market (suppliers and end-users) on the similarities and differences between the two processes;
    • Educate and encourage the investor community to keep increasing their interest and investment;
    • Expedite the commercialization process.

    Distribution will include:

    • BPI's 30,010 qualified readers;
    • Delegates attending ISCT's 2011 Annual Meeting (included in all delegate bags)
    • Delegates attending ESACT 2011 (Chair drop at the Cell Therapy Plenary Session)
    • INTERPHEX 2011 Cell Therapy Roundtable (VIP Invitations, 200 attendees, produced by BPI)
    • BIO 2011 International Convention (BioProcess Theatre - Cell Therapy track)

    If you are interested in advertising, click here for more info.

    While the content for this issue is now being finalized, it you are interested in contributing something to BPI, we are looking for more cell therapy related content. As the cell therapy representative on BPI's advisory board I would be happy to champion it through submission.

    Cheers.

    --Lee

    To Read More: Cell Therapies: Commercializing a New Class of Biopharmaceuticals
    categoriaRegenerative Medicine commentoComments Off on Cell Therapies: Commercializing a New Class of Biopharmaceuticals | dataFebruary 12th, 2011
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    The LinkedIn Cell Therapy Industry Group – 1,000 members strong

    By Dr. Matthew Watson

    As some of you may know, much of my recent social media energy has been spent on LinkedIn rather than blogging. This was not a conscious decision but I will admit to finding the immediacy and interconnectivity of the LinkedIn/Twitter combo to be more seductive of my limited time than the more laborious and seemingly more unidirectional facets of blogging. I'm still working on a return to more diligent and regular blogging - we'll see how that goes.

    In any event, today's blog entry is ironically about the very thing which has replaced my blogging in many ways for the interim: the LinkedIn Cell Therapy Industry Group which I founded in July 2008 (about the same time as I launched this blog).
    Primarily due to the outstanding participation of great members, the group has turned out to be what I had hoped would be and I believe has become a fairly valuable resource for those in or interested in the cell therapy industry.
    The group grew exponentially throughout 2010 and we are proud today to announce our 1,000th member. Without his knowledge, Luc Gervais today became the 1,000th member of the LinkedIn Cell Therapy Industry Group.
    Luc Gervais lists himself on LinkedIn as a "Technologist Entrepreneur" but is also a Researcher at IBM Research, Zurich Research Laboratory in addition to being a researcher at the University Hospital Basel.
    He was recently involved in the development of IBM's novel, microfluidic "lab on a chip" technology that uses capillary action to create a potential one-step diagnostic tool, and which could ultimately test for a wide range of diseases and viruses. The chip requires only a small drop of blood, which it draws through tiny channels within the device. The blood reacts with different disease markers to provide accurate diagnoses in about 15 minutes.
    Luc represents what I believe is one of the most exciting signs of development in and maturation of the cell therapy industry. Luc's career has included being a Game Developer at Unlikely Games, a Computational Chemistry Developer at Boehringer Ingelheim Pharmaceuticals, and a Quality Assurance Specialist at Steltor. On LinkedIn, he lists "regenerative medicine" as one of his interests.
    People with the kind of experience Luc possesses are bringing a world of scientific, technical, and commercial expertise to regenerative medicine and cell therapy from outside the sector. This promises to revolutionize the way we think about, develop, and apply our technologies.
    Luc and others like him who are exploding into the regenerative medicine and cell therapy field bring with them the potential for interdisciplinary exploration, the opportunity to draw from lessons already learned in other sectors, and the chance to view our field not just in terms of the incredible potential for new therapeutics which cell therapy represents but how that fits into the broader world in which cell therapy is growing up. A world that includes phenomenal advancements in personalized medicine, diagnostics, theranostics, biomarkers, bioinformatics, the ability to access and interpret personal genomics data, etc.
    I have yet to speak to Luc (this was all posted from publicly available information) but I'm hoping to bring you an interview of him shortly not because being the 1,000th member of the LinkedIn Cell Therapy Industry Group is deserving of any particular attention (and certainly will not rank in his list of accomplishments I'm sure) but because I'm curious about what Luc represents.
    Stay tuned....
    --Lee

    To Read More: The LinkedIn Cell Therapy Industry Group – 1,000 members strong
    categoriaRegenerative Medicine commentoComments Off on The LinkedIn Cell Therapy Industry Group – 1,000 members strong | dataJanuary 15th, 2011
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    Careers in cell therapy & regenerative medicine

    By Dr. Matthew Watson


    As you will see just below, this post falls under the category of a thinly veiled and somewhat shameless plug which I hope you'll tolerate if I'm transparent about it upfront and I add what may be a little useful commentary along the way.

    I won't have to convince "regular" readers of this "irregular" blog that regenerative medicine is one of the fastest growing life science sectors.

    Given the pace of the industry's growth, the amount of public money being "invested" in the sector, and the general enthusiasm that the science generates, it is no surprise that is increasingly an area of interest for those seeking a new career path.

    One commonly held misconception is that career opportunities in the sector are limited largely to those in early-stage research.

    While it is true that the industry is still in the nascent stages of commercial development compared to other more mature segments in the broader biopharmaceutical industry, careers in regenerative medicine span the full research and commercial spectrum from pre-clinical to clinical research in both academic and corporate settings, manufacturing, regulatory, quality, operations, logistics, finance, business development, marketing, sales, communications, and executive management.

    As has been discussed here in the past, most analysts agree that in 2008 the industry passed the $1 billion mark in annual revenue generated from sale of approved therapeutic products falling under the regenerative medicine category (see here for more in-depth analysis).

    Furthermore, most large multinational life science companies are now investing heavily into the sector on the promise that it will revolutionize healthcare in the coming decades.

    I believe the 2008 decision by Pfizer to create a substantial new Regenerative Medicine division will be seen as a seminal turning point in corporate perspectives on the industry. Since that point most life sciences have begun investing in regenerative medicine strategies and the trend has even leaked outside of life science to companies such as Google Ventures which has informally identified regenerative medicine as one of their primary areas of interest when looking at companies in which to invest.

    Most major universities have now created both academic and research programs dedicated to the emerging field of regenerative medicine such that there is a new class of graduates now emerging in both scientific and commercial disciplines with regenerative medicine as a primary focus.

    However, one of the difficulties with finding a career in regenerative medicine is that it is comprised of what has heretofore been fairly discrete disciplines of stem cell biology, pharmaceutical sciences, biotechnology, tissue engineering, stem cell transplantation, device technologies, cell therapies, etc.

    Furthermore, most recruiters and online job sites have very little experience or focus on the industry making recruiting difficult for positions in regenerative medicine companies or departments. The task is further complicated by the fact that the candidate pool is small and diversely spread over a multitude of disciplines, centers, and a multitude of small companies.

    Finally there are only now emerging industry publications, organizations, online communities, and websites that truly represent the broad spectrum of regenerative medicine and that can be used as central resources for recruiting.

    All this is why my partners and I saw the need for an online recruiting tool that really focused on regenerative medicine in a way that would support the industry's maturation. What we wanted to bring was a very simple solution that would create as much value as possible for those looking to recruit the right talent into their regenerative medicine efforts. This was the genesis behind RegenerativeMedicineJobs.com.

    (here comes the shameless part....)

    About RMJ

    At RegenerativeMedicineJobs.com, we believe the growth trajectory of this industry requires a specialized, online recruiting system – one that is more than just a website for posting jobs but also plugged into the large and diverse network of associations, publications, and social media that serves this industry.

    RegenerativeMedicineJobs.com is a specialized online job board focused on recruiting for positions in the rapidly growing field of regenerative medicine. The site is simple to use and focused in scope. But this is more than just another website.

    RMJ has developed a sophisticated system designed to leverage our extensive network in RM to ensure every post gets maximum attention and attracts the best candidates.

    Traffic is pulled to the site and job postings are pushed to a targeted audience by focused social media and marketing campaigns. This is the latest way to recruit for any position in a regenerative medicine department, division, or company. This is regenerative medicine recruiting with a focus.

    RegenerativeMedicineJobs.com (RMJ) is a collaboration between CTG Consulting, Co. and Pencilneck Software, Co. The personnel behind this collaboration created Cell Therapy News, Cell Therapy Blog, the LinkedIn Cell Therapy Industry Group, and have produced over a dozen organizational or event-based websites in the cell therapy, stem cell, and regenerative medicine space.

    The site has been designed to be simple and intuitive with only one goal in mind: to be the best online tool for recruiting personnel into positions in the regenerative medicine industry.

    We don't offer resume posting, career advice, assistance building your CV, hand holding, shoulders to cry on, or job postings outside of regenmed. We believe in the value of specialization.

    The site is a listing of regenerative medicine jobs (period). All types of jobs for all types of organizations but only regenmed job (period).

    We have only one rule: the jobs posted must be for a position within the regenerative medicine space. We define regenerative medicine as anything involving cell therapy, stem cells as therapies or tools for discovery or toxicity testing, or non-cell based programs/products (e.g., small molecules, biologics, devices, etc) used to replace or regenerate cells, tissues, or organs to restore, repair, or establish normal function.

    The site has a topical niche with no regional focus or bias. This is meant to be a global tool equally useful for a company or university in Poland, Taiwan, Brazil, the United States, etc.

    The site is also meant to be useful for recruiting any positions scientific or commercial, business or technical, executive or laborer.

    *

    We hope you find it useful.

    We hope it contributes to the industry's growth and maturation.

    We hope you will provide us feedback on what we're doing well and what we can do better.



    To Read More: Careers in cell therapy & regenerative medicine
    categoriaRegenerative Medicine commentoComments Off on Careers in cell therapy & regenerative medicine | dataOctober 11th, 2010
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    FDA files injunction again Regenerative Sciences citing Regenexx violates regulations

    By Dr. Matthew Watson

    For those of you who follow this blog, you'll imagine my surprise to wake up the morning to the following announcement from the FDA:
    _____________________________________________________________
    FDA NEWS RELEASE
    For Immediate Release: August 6, 2010
    Media Inquiries: Shelly Burgess, 301-796-4651, shelly.burgess@fda.hhs.gov
    Consumer Inquiries: 888-INFO-FDA
    FDA Seeks Injunction Against Colorado Manufacturer of Cultured Cell Product
    Violations of current good manufacturing practice and labeling requirements cited
    The U.S. Food and Drug Administration is seeking an injunction in federal court against Regenerative Sciences LLC, of Broomfield, Colo., citing violations of current good manufacturing practice (cGMP) that cause its cultured cell product to be adulterated. The product is also misbranded due to the lack of adequate directions for use and the failure to bear the “Rx only” symbol.
    The company’s cultured cell product is derived from a patient’s bone marrow or fluid surrounding the patient’s joints (synovial fluid). The cells are grown, processed, and mixed with drug products outside the body before being injected back into the patient.
    Regenerative Sciences’ cultured cell product is not approved by the FDA, and no adequate and well-controlled studies have been done to demonstrate its safety or effectiveness for any indication.
    “FDA recognizes the importance of the development of novel and promising new therapies,” said Karen Midthun, M.D., acting director of FDA’s Center for Biologics Evaluation and Research. “However, when companies like Regenerative Sciences fail to comply with FDA laws and regulations, they put the public’s health at risk.”
    The complaint for the injunction was filed Aug. 6, 2010, by the Justice Department on behalf of the FDA in the U.S. District Court for the District of Columbia, against Regenerative Sciences and three of its employees, Christopher J. Centeno, M.D., John R. Schultz, M.D., and Michelle R. Cheever. The injunction would permanently prevent the company and cited individuals from adulterating and misbranding the cultured cell product while the product, or one or more of its components, is held for sale after shipment in interstate commerce.
    Regenerative Sciences has agreed to cease production of the cultured cell product while the case is pending.
    The FDA warned Regenerative Sciences about its cGMP violations as recently as June 2010. The company failed to make sufficient corrections, and the conduct of the individuals cited in the complaint demonstrates refusal to comply with the law.

    To Read More: FDA files injunction again Regenerative Sciences citing Regenexx violates regulations
    categoriaRegenerative Medicine commentoComments Off on FDA files injunction again Regenerative Sciences citing Regenexx violates regulations | dataAugust 12th, 2010
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    The changing face of PR and why it matters to regenmed

    By Dr. Matthew Watson

    This may seem a little off-topic for those who don't know me but for those of you who do, you'll know this is a little mix of many of my primary loves - cell therapy/regenerative medicine, communications/public relations, social media, and all things internet/technology.

    This is a reprint (with permission) of an article I was invited to write for the 2009 World Stem Cell Report.

    ______________________________________________________________________

    If you’re breathing, you’re in PR [1]
    Some Do’s and Don’ts and 2.0s for orgs/co’s in cell therapy & ReGEN

    You may be one of them. The scientist who can’t get the job or promotion you want. The company not securing customers fast enough. The investigator not finding collaborators. The company not finding quality employees. The company failing to secure much needed investment. The big company not effectively penetrating new, niche markets like cell therapy-regenerative medicine. The executive or investigator not getting the speaking invites to create the buzz needed to help move things forward.

    Whatever the challenge, consider a healthy dose of marcom. [2]

    Ok, I’m biased and it certainly isn’t a single panacea for all these problems, but seriously. A good profile (personal or corporate) – built from a solid reputation and strong relationship network – can help address almost any challenge you or your company is facing.

    INTRODUCTION

    I know. You don’t believe me. Humor me and answer this one question. If you could double the number of people that know about your company or technology, the number of people that learn, hear, or read something about your research, product or service in the average 30–day period, and/or the number of people that interact with one of your employees or colleagues every week, would that – on the whole – likely help or hinder you in addressing at least some of the primary challenges you are currently facing?

    See where I’m going with this? Even if your challenges are deeply scientific or technical, is it possible to imagine that if only you knew the right person …?

    Your existence depends on human interaction. Your success depends on how well you interact and the impressions you make. The impressions are not solely or even primarily driven by the brilliance of your technology or the compelling weight of your data. This is likely more true than you may think. For those of you who have read Malcolm Gladwell’s book, Blink, you will appreciate just how much people’s perceptions are influenced not by logic, data, or comprehensive consideration but by the judgments of the subconscious in a matter of seconds.

    The PR (public relations) I discuss in the following pages is not spin and it’s not advertising. It’s about establishing and maintaining relationships that result in profile among your various kinds of constituents be they your collaborators, peers, investors, employers, employees, customers, or potential strategic partners.

    Successful people and companies use effective PR to create relationships, turn them into interactive networks, and then leverage them to help solve the challenges they face.

    This has always been true. What are so radically changing today are the tools which people and companies can now employ to conduct effective PR, create relationships, and build networks all at a fraction of the cost, with greater returns, and performed by individuals or small companies not previously capable of affecting global impact. These tools are, of course, online.

    A successful strategy for PR is now increasingly determined by how well online tools are employed to engage in conversation, create relationships, and build profile.

    As it turns out, there are, in my opinion, too few people in this industry who, as yet, understand the power of PR and fewer still who understand the potential of web2.0 to change their fate.

    What I will share with you in the next couple pages is:

    1. why if you’re breathing, you’re in PR - if you can’t avoid it (and I don’t recommend you try), you should learn to embrace it;

    2. the cost of dodging the embrace; and

    3. how you can embrace it.

    POINT #1: YOU’RE IN PR. REALLY.

    There are a zillion reasons why you underestimate the importance of PR in your career or to your company. There are a zillion other reasons why you think online social media is irrelevant to what you do. Let’s look at five.

    1. My company is a research-based, early-stage company conserving burn-rate, with few employees, and nothing to sell. Ergo we don’t have a need for PR stuff like marketing, communications, or profile building.

    There is nothing new but certainly nothing outdated in the adage: Everyone is selling something.

    Don’t pretend you have no one to impress, you have nothing to sell, or that content is the only thing that impresses in the science business. If you thing you have nothing to market, you just haven’t figured out what it is yet.

    Are you looking to recruit quality people? Do you anticipate needing to find and impress new investors sometime this year or next? Would you be interested in engaging in discussions from parties potentially interested in licensing or acquiring some of your IP? Are you looking to impress potential academic or corporate collaborators? Do or will you need to recruit patients for a clinical trial? Do you need to start educating doctors about the potential of your therapeutic or engage them in giving advice about your trial or product? Would you benefit from increased government funding for cell therapy and regenerative medicine? Do you believe there are policy changes that could be made that would benefit this industry?

    A better profile and a wider network of contacts can’t help but help you address these needs. If you have anyone to impress (and everyone does), then you need to be talking with them. Will a bigger and better profile hurt your efforts to find good employees, seduce investors, recruit patients, identify collaborators, secure partners, etc? It’s hard to imagine how.

    At the very minimum, as a member of the life sciences industry you have an obligation to get out of your lab and communicate to the world around you about the science you are so passionate about. As President Obama encouraged in his address to the April 27, 2009 National Academy of Sciences [3], we all have a duty to do a better job of engaging the people around us in a discussion about the importance of science and the science we’re doing.

    2. My company is a big, public, multi-national company with departments that handle marketing, communications, PR, and advertising. Ergo I don’t need to pay attention to these things because they do that.

    Firstly, unless you have tenure, chances are between now and retirement you may want/need/be recruited for another job. A strong, personal “brand” (profile) is certainly a huge asset in terms of ensuring you are successful in finding that job and negotiating the right remuneration.

    Secondly, if you are ever let out of the lab/office or given internet access, you have the capacity to (and likely already) influence your company’s profile and brand. You are either complementing or distracting from your company’s primary messages and brand.

    Thirdly, if you care about your company’s success, don’t assume your company’s department or external marcom firm is doing what needs to be done. Investor relations, marketing and communications firms or departments may be good at investor relations, marketing, and communications but that doesn’t necessarily mean they are good at the kind of profile building that is going to attract and support meaningful business development opportunities or potential investigators, customers, licensees, collaborators, employees, or clinical trial patients.

    Finally, too many big companies haven’t yet figured out how or how to successfully use Social Media. If you’re using Social Media (e.g., Facebook, Twitter, FriendFeed, Digg, etc), you might be able to play a leadership role in being the face of your company on these platforms – just don’t do it without permission!

    3. My company is a science-based company with a technical sale to scientists who are not receptive to glitzy marketing, advertising, PR, and-the-like. Ergo there is little ROI on spending money on these kinds of things. We don’t “do” marketing.

    You may be in a company that believes putting out press releases is an unnecessary distraction from the business of science; or the company that believes that communications is something done only big companies, public companies, stock hustlers, or companies with something to sell.

    Wrong. First you’re working on the assumption that all marketing looks like a drug ad.

    Second your target audience is human. Humans are susceptible to having their perceptions influenced via all the usual means. Even if you don’t want to look at it so crassly, you would have to agree they are susceptible to forming more positive impressions of your company or product if information about it is available, packaged nicely, and easily accessible. You would also have to agree that any impression or sale is easier to make when there is a personal relationship/contact.

    Your network is one of your most valuable assets to you and to your company. Building a global network and industry profile is marketing under what ever name you want to call it.

    4. All this web 2.0 and social media stuff is for tech geeks and kids. They are not my target market and don’t have a critical mass of the kinds of people I need to building profile among.

    Let’s assume for a moment, this is true. If you’re looking to be an attractive employer to potential, young employees you may want to reconsider.

    Secondly, young people grow up and become industry leaders and executives. Is it too early to start shaping their perceptions?

    Thirdly, there is the occasional executive that is actually occasionally influenced by input from their younger employees. I know – it’s crazy!

    Furthermore, it’s not true. For example, I have a very high percentage of c-level executives on my LinkedIn Cell Therapy Industry Group, Jim Till who proved the existence of the stem cell in his ground-breaking nature paper in 1963, is an active poster on Twitter, and the average age of social media users is much older than you believe - check your data.

    5. Twitter-schmitter. Today’s social media sites are tomorrow’s relics. They’re all hype when they launch and then they fall out of favor and are no longer useful. I’m a serious business professional with no time or need for these gimmicks that have no real business applications or executives like me on them.

    If you think LinkedIn, Twitter, or Facebook (my three favorite and most used social media sites) are not going to be around and successful for long enough to have a real impact on your business, you’re definitely one to bet against the odds.

    If you think serious business is not being done and useful business connections being made on these sites (see the last paragraph in the point above), you’re dead wrong. I’ve found customers, employees, and collaborators for my customers using social media in addition to getting them speaking engagements and highlighted in publications.

    The point? Everyone has some kind of PR (marketing, communications, profile building, networking, advertising, community participation, publishing, speaking, etc) to do. Your potential audience, network, and impact are all much bigger than likely believe to be true.

    My guess? You have more than likely significantly underestimated the value of engaging in PR. And, you have likely significantly overestimated the time and financial cost of engaging in PR.

    That brings me to Point #2.

    POINT #2: THERE IS A REAL COST TO YOU AND YOUR COMPANY IN PRETENDING YOU DON’T DO OR NEED TO DO PR.

    All I should really have to do here is refer you back to point #1. The benefits of engaging in PR are the costs for not. Nevertheless, I will briefly expound.

    Your next job, employee, collaboration, investor, customer, patient, etc will be influenced by the profile of you and/or your company. While your CV – comprised of its peer-reviewed publications, presentations, and collegial references – is still an important driver in people’s perceptions in our industry what often makes someone or some company stand out among the others (perhaps even imperceptibly) is the overall profile one has created. One’s profile is increasingly judged online.

    That brings me to revise Point #2 to the following:

    There is a real cost to you and your company in pretending you can/should ignore the PR power of web2.0’s social media.

    I’m going to steal now from my October 2008 blog posting on the subject. [4]

    Only a few minutes ago (relatively speaking in the passage of time) most companies didn't think they needed to have a website or a domain name strategy. This seems as foolish now as ignoring social medial will seem in a few minutes from now.

    In 1995 - a mere 14 years ago - I was a young associate in a downtown law firm making a pitch to a skeptical management committee at a weekend retreat that the firm needed a website and an email "system". It was not an easy sell. They "knew" lawyers would never send their own emails and certainly not use it to communicate with clients. Similarly they were convinced a website would likely not bring in any new clients and existing clients would not likely find a website useful.

    How quickly they were proven wrong.

    We are at a similar technological threshold. Most companies are ignoring the importance of "social media" to their corporate strategy. Most consider using social media an unnecessary "luxury" in exactly the same way websites, domain names, and email were considered by people in their positions only a few years ago. They do not understand how social media is already changing the way they do business, the importance of staking position/profile early, nor the pain they will feel in the very near future for having failed to be proactive in building and leveraging online communities.

    This is very understandable. In times of fiscal constraint one tends to focus on that which hurt you yesterday or causes you the most pain today. Nonetheless, the companies that will succeed tomorrow are looking at tomorrow now and preparing for it. Tomorrow's mainstream business activities involve social media as much as today's involves website and email.

    For those thinking that this will be true only for businesses that deal direct with the retail consumer, they’re dead wrong. If you have a constituency that you need to keep informed about and actively engaged in your company, then this applies to you. Whether its business (B2B) customers, investors, media, patients, recruits, employees, or collaborators, they are online and engaging in social media. If you're not there, they're listening to and engaging with someone else.

    LinkedIn now has 42 million members with 1,945,047 members who identify themselves in biotech, pharma, or healthcare. Some 300,000+ of them are in biotechnology. There are 293 biotechnology groups in LinkedIn and 375 biotechnology groups on Facebook. The LinkedIn cell therapy industry group has 400+ members engaging in active discussions and exchanges of information in the public forum and using the platform to make and strengthen connections offline.

    Bottom line? There are so many different kinds of social media out there that can be used to reach so many different kinds of audiences at a fraction of the cost of traditional media that it's just good business to figure out how to use it to your advantage. In fact, you may be feeling the pain of ignoring it already and just don't know it...

    Which brings me to point #3.

    POINT #3: WEB 2.0 HAS PUT THE ABILITY TO DO EFFECTIVE, LOW-COST PR IN YOUR HANDS. THERE ARE NO MORE GATEKEEPERS.

    The internet has blown the sense of community up and redefined it. Gone are the days when it was feared the web would destroy “community”. Instead web 2.0 is all about creating more and larger communities than we ever thought possible eliminating all kinds of barriers and cutting across all kinds of definitions.

    It used to be (before web 2.0) that corporate profile, marketing, communications, PR, advertising was all quite carefully controlled by managing the one-way push of information to the open receptacles we called “readers” or “audience”. If you wanted a global profile, you needed a global PR firm.

    Just like the internet has taken the magic out of the business of travel agents, web 2.0 has forever changed the grip that marketing, communications and PR firms had on the business of managing global corporate profiles. Similarly, a global profile is no longer only the purview of companies sufficiently large to retain the services of such firms or have big, internal PR departments.

    Just as the information on how to build long-range missiles is no longer the exclusive domain of rocket scientists, with the empowering tools of web 2.0, successful marcom can now be effectively done by subject-matter experts who are not big-budget marcom professionals.

    It’s why newspaper are dying and bloggers are thriving. It’s why small companies are building niche profiles that beat out their Fortune 500 competitors. It’s why startups can have a global profile in weeks on next-to-no budget.

    All of that is a rather lengthy introduction to primary point of this article:

    In this new world of interconnectedness and easy-to-access information, it is easier than ever for everyone and anyone to influence the profile of a company and what it is offering to the world. That’s the good news. The bad news is that you are expected to. All of which leads to one not-so-original observation of the impact of web 2.0 on companies.

    If you’re breathing with an internet connection, you can and should have significant and immediate impact on the awareness and perceptions of your company among people around the globe.

    If you’re not creating global awareness and positive perceptions, it may be assumed you’re terminally short of breath.

    Like it or not. You can’t help it and you certainly can’t avoid it. And the people forming these perceptions are not just the potential customers of companies with something to sell – they’re current or potential investors, partners, collaborators, patients, purchasers, journalists, regulators, policy-makers, employees, employers, etc.

    LIGHTS, CAMERA, ACTION

    So what can you do to begin leveraging the power of the PR tools at your fingertips and turn a bungalow profile into a chateau; a respectable list of contacts into a vibrant, enviable network of relationships?

    10 Ways to a Better Profile for RegenMed and Cell Therapy Organizations

    I’ll start with some rules that apply regardless of the PR platform you’re employing (e.g., traditional ones like conference presentations or journal publications or web2.0 ones like blogging or social networking).

    1. You need to have a communications strategy and then mandate and empower your people to communicate your message/story to your audiences.

    This doesn’t have to be complicated. It’s about defining your primary/secondary audiences, messages, keywords, and tactics to accomplish your primary/secondary goals.

    2. Talk about what you’ve done not about what you’re going to do.

    It is very rare that news about what you are going to do is really newsworthy. Don’t be tempted to put out a press release that’s not news just because you haven’t put one out in awhile. For example, a press release that your executive is going to speak at a conference is not news unless they are presenting new data or they are the keynote at the biggest conference of the year. That doesn’t mean you can’t post it on your website or let your network know in other ways but the press release should be used judiciously.

    3. Make sure you tell the market story not just the science story. Similarly, be clear about what problem your technology solves not just how brilliant it is.

    4. Be upfront about the limitations of your product/service. Don’t try to pretend you have the perfect product/service. Don’t try to be everything to everyone – defining who you are (your company and its product/service profile) is just as much what about what it is than what it is not.

    5. Avoid the temptation to promise when you will do something by (e.g., begin or end your trial, submit your IND, launch your product, etc) unless it’s the subject of a contractual obligation. There are too many unknowns that may prevent you from meeting the expectation you created. The ensuing disappointment will hurt your profile and perceptions of your company and its product/service and management.

    6. Don’t over communicate your regulatory interactions with the FDA – this is one area where the less said is often better.

    7. Wait on that hiring announcements just a little while. It’s critically damaging to put out a press release that now has a very, long online life outside of your control announcing the hire of a key executive only to have him/her not last beyond the first few weeks.

    8. Understand that communications, marketing and profile building are very different from advertising. Act accordingly.

    9. Value the untapped potential of your existing customers and prospects to spread your gospel.

    One of the real discoveries from social media is the alarming willingness of people to zealously proselytize on behalf of companies and products win which they believe. Find ways to actively engage them in doing so. [5]

    10. Engage online.

    The 8-step Plan to Enhance your Online Profile and Network

    1. SEO.

    If you’ve decided your profile matters, then your online profile really matters. The fastest way to an impressive online profile is Search Engine Optimization. There are a lot of tricks up the SEO tricksters sleeve and I’m not about to give them all away but suffice it to say that SEO is a multi-pronged attack and there are many arrows in the quiver each with a different use to the same end: higher rankings on the major search engines = increased traffic to the website = greater profile = whatever your end goal is (sales, investors, collaborations, changing the world, etc).

    2. Listen & Monitor.

    Whether you employ Google Alerts, Twitter search tools, or simple subscriptions to topical news services or blogs, there is a lot of content and discussions being added to the internet every day. You will likely be amazed how much (or how shockingly little) your company and product/service is being discussed and who is behind the discussions. Listening to and reading what’s going on the internet will get you intel about your competitors, new ideas for potential products or studies, new leads for potential collaborators or customers, etc.

    3. Follow/Subscribe.

    Find your favorite content providers, writers, resources, publications, etc and follow or subscribe to the content their pushing out and discussions they are leading.

    4. Join

    There’s no harm in joining. People join for all different reasons. There’s no problem in being an observer. In fact it’s likely best you just observe for a while after you join to assess the flavor of the group and discussion so when you do participate you don’t offend.

    5. Participate

    Once you’re comfortable, start to participate. Engage in discussion, exchange information, and above all make sure you are providing value to the other participants not just talking about yourself. This will be punished.

    6. Connect (yourself and others)

    There’s a reason why these are called online networks or communities. Use them to connect with people not just for the sake of building a bigger ‘friends’ list but because you have something in common and want to begin building a relationship.

    7. Create

    Be original. Create something of value you can share. That might just be facilitating a channel in which 3rd party information is exchanged. It might be you writing original content. It might be facilitating introductions.

    8. Enjoy and don’t offend.

    Most importantly find the online networks that you enjoy, that give something of value to you, then contribute value in return and ensure you don’t offend the rules. Nothing slaps harder than an online, viral slap from a community you’ve offended online. [6]

    CONCLUSION

    Don’t pretend you have no one to impress. Everyone needs to engage in PR.

    Even in the business of science, man cannot live on data alone. You need more than just solid content to affect the kind of impressions that will be critical to your success. Don’t let your data or career speak for itself – it can’t talk.

    Web 2.0 has made it exponentially easier – if not expected - to engage in PR and profile building.

    What’s missing from this article is the web 2.0 tools I’d recommend you use to put the 8-step plan in place. For that, watch here for further blog postings on the subject. See what I did there?



    [1] In this article, I use “PR” not to mean “public relations” as you may have come to understand it used but rather simply to mean any kind of relating to the public via marketing, communications, writing, profile building, networking, advertising, community participation, publishing, speaking, proselytizing, lobbying, etc. It’s a not-so-dirty little secret that we all do it in one form or another or several all at once.


    [2] Marketing & communications


    [3] See http://www.youtube.com/watch?v=k5-MgZD5IMc at around the 30 minute mark.

    [4] Oh, I haven’t mentioned my blog? It’s at http://www.celltherapyblog.com/. Of course if you are a subscriber, this will be a refresher. For those who haven’t read this on my blog, you would have benefited from reading these same thoughts back in October 2008 if you had been a blog subscriber, or if you followed me on Twitter (I’m @celltherapy) or if you were connected with me on LinkedIn (http://www.linkedin.com/in/celltherapy). I’m just saying…


    [5] One the flip side these same people will use these same tools to punish companies mercilessly for offending them or the rules of social media.


    [6] Just ask Johnson & Johnson about the power of mommy bloggers. Look it up.



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