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Friday, November 21, 2008

Cell Therapy HiLites 2008-11-21

This is a week in which 2 of GEN's top 7 News of the Week stories were about regenerative medicine and they weren't even the two big financing events. Not bad.

Good news for Tissue Genesis Inc and their Commercialization Consortium partners Bioheart and VetStem. Effective immediately, the Tissue Genesis Cell Isolation System will be available to the European marketplace.

In a piece about biotech survival, Luke Timmerman, now of Xconomy.com, reports that according to an October report by Eun Yang, an analyst with Jeffries & Co., "about half of the 248 unprofitable biotechs that are publicly traded have less than a year’s worth of cash on hand".

Dr. Michael D. West announced an open letter to President-Elect Obama signed by 22 scientists and economists (including 19 Nobel laureates), urging the new Administration to make regenerative medicine, specifically, the cure of human disease, a national priority, similar in scope to the Kennedy Administration’s commitment to land a man on the moon within that decade.

Aldagen announced positive phase 1/2 results for ALD-301 (an autologous, bone marrow-derived stem cell population expressing high levels of ALDH) for critical limb ischemia.

The story everyone loved this week was the new trachea built from autologous stem cells and effectively transplanted in Spain. Chalk one up for more proof-of-principle but more consternation on the "business models" side of things. Researchers and surgeons from Britain, Italy and Spain collaborated to grow tissue from Ms Castillo’s own bone marrow stem cells, using them to fashion the new bronchus – a branch of the windpipe. Details of the transplant, performed by Paolo Macchiarini, at the Hospital Clinic of Barcelona, are published online in The Lancet and discussed in accompanying commentary. First a section of trachea was taken from a donor and stripped of cells that could cause an immune reaction, leaving a grey trunk of connective tissue. Stem cells were then taken from Ms Castillo’s bone marrow and grown in Professor Birchall’s laboratory to cover the 7cm graft. It was then “seeded” with the new cells using a process developed in Milan. Finally the trachea, covered in cartilage and lined with epithelial cells, was cut to shape and fitted. The researchers said that the surgery could help some patients in Britain but admitted that the procedure was too expensive to be widely available. They are seeking EU funding and commercial sponsors for trials to create and transplant a larynx, an operation that could be more cost-effective.

The two stories I loved the most this week were finance-related.

Last week Stem Cells Inc. announced its intention to raise $20M gross and this week it closed the deal netting $18.5 million from institutional investors.

Not to be outdone, Tengion raised $21 million in a second close of its Series C adding Safeguard Scientific to its heady roster of investors.

What money does exist for investing in cell therapies appears to be increasingly focused on a handful of companies that have demonstrated a somewhat mysterious capability to ease money from investors. Stem Cells, Inc and Tengion are two of the best along with MolMed in Italy which netted around $76M in an IPO earlier this year. Osiris has also done well on the public markets but also through the undying commitment of Peter Friedli and timely deal-making. Notable mention should also go to Aldagen (despite them having to recently pull a planned ~$80M IPO), Novocell, Geron, and Dendreon who all have done well in the past with investors.

This trend for capital congregation is nothing new. It certainly supports popular notions that either investors all think the same or they are lemming-like in their movements. It certainly does not bode well for the plethora of companies out there that are on the brink of burnout despite having seemingly strong management and data but do not seem to have that same type of investor-attracting charisma as the likes of Weissman/McGlynn, Nichtberger, Bordignon, Mills, Lewis, Okarma, or Gold.

At the end of the day, a company's success almost always comes down to its rainmaker. This is not a "if-you-build-it-they-will-come" type of industry. Great science, technical prowess, manufacturing experience, or even business acumen will not get companies over the threshold without that someone with that somewhat magic prowess to relieve investors of their money. Many cell therapy companies are now learning that lesson.

In the interim, from a mere objective point-of-view I could argue it's not a bad thing that investors focus precious resources on a few companies' products, get them approved, and get some fresh upticks in the "precedent" column for cell therapies and the new therapeutics and business models they represent. Like lawyers - and perhaps its because there are so many law degrees in the halls of VC firms - nothing makes them more comfortable than precendent.

That's just the way I saw cell therapy this week...

Monday, November 17, 2008

How mature is the cell therapy industry? Do we know?

Survey
The week of November 17, 2008 I posed the following one-question survey:

How many phase III or pivotal industry-sponsored cell or gene therapy clinical trials do you believe are currently ongoing in the world?

to members of the following groups:
  • those who follow me on Twitter (currently a group 43 individuals sufficiently interested in cell therapy to follow my postings);
  • my connections on LinkedIn (a group of 188 professionals in or interested in the cell therapy sector);
  • the 90 or so members of the LinkedIn Cell Therapy Industry Group;
  • readers of this Blog; and
  • the roughly 55 members of ISCT's cell therapy commercialization committee.
There was no control on multiple responses and no restriction on participation. Participants were forced to choose only one answer among responses ranging from 1-5 to 50+ in increments of 5 (e.g., 1-5, 5-10, etc).

Data, Assumptions, & Observations
There have been 18 responses to-date.

If we assume that only members of the groups listed above participated in the survey, then it is reasonable to characterize the participants, at the very least, as informed participants who know more about the cell therapy industry than the average person. I will also assume that participants only voted once.

Based on these assumptions, the data leads to one simple, overarching observation:
There is significant divergence of perception (and perhaps misconception) about the maturity of the cell therapy industry even among those in and/or informed about the sector.
My database tells me there are 33 industry-sponsored cell or cell-mediated gene therapy trials currently underway around the world.

45% of survey participants answered fewer than 15 to the survey question; 33% indicated more than 45. In fact, only 1 participant (6%) do-date has chosen the category of 30-35.

Analysis
Admittedly this is an unscientific and informal survey. Furthermore there is likely some margin of error to account for different interpretations of what "industry-sponsored" means and/or what types of products should be included/excluded as "cell or gene therapies" since none of these terms were defined for participants.

Certainly in my day-to-day dialogue with people in the industry, the typical opinion is that there are but a handful of late-stage cell therapy trials (far fewer than what my data tells me actually exists). Having said that, when I asked a colleague in the industry yesterday to respond verbally to the survey question, they said "350" ( and then pulled back to say "maybe 200").

There is no doubt my data likely lacks a material number of industry-sponsored, phase III or pivotal gene therapy trials - given that the focus of my database is cell therapy and thus typically only includes cell-mediated gene therapies. This exposes a weakness in comparing the survey results to my data in that the survey related to "cell or gene therapy" while my data likely only reflects a small subset of gene therapy trials.

There is also without doubt more than 33 cell therapy or cell-mediated gene therapy clinical trials currently underway in the world with many of the indicia of a "late-stage" trial if one includes what is happening in markets where such trials or products are currently unregulated. I have not included these in my data because there is no mechanism for identifying whether such trials are, indeed, phase III or pivotal as there is no mechanism as yet in those markets for formal review or approval of these trials and/or products.

Nevertheless, what conclusions can/should be drawn?
  • There is much divergence of perception on the maturity of the cell/gene therapy industry even amongst those in the sector.
  • Even if my data is inaccurate, compared to whatever the real number is there is a significant % of industry professionals who are suffering from significant misconceptions about the true number of late-stage industry-sponsored trials in the sector.
  • Good data on the state of the cell/gene therapy industry is not readily accessible.
  • If those in the industry lack consensus on the state of the sector, it is no wonder investors and others outside the industry have mixed opinions on the matter.
  • We need to do a much better job as a sector in gathering and communicating solid data on the commercial side of the cell/gene therapy industry.
For those interested in the data, here's how it looks:


Industry-Sponsored Cell or Gene Therapy Phase III or Pivotal Clincial Trials (Nov 2008) - DocStoc

Coming soon...a similar discussion on the number of cell therapy products currently commercially available around the world.

In the interim, if any of you have data relevant to this discussion, please share...


Friday, November 14, 2008

Cell Therapy HiLites 2008-11-14

Here's what caught my attention this week:

Celgene received FDA clearance to proceed with its placenta-derived stem cell clinical ttudy - a phase I, multi-center trial for moderate-to-severe Crohn's disease.

MiscroIslet filed for Chapter 11 bankruptcy protection but expects to restructure as a private company, continue operations, and file its first IND by year's end for an encapsulated porcine-islet product to treat diabetes.

Celladon released promising phase I results for the first gene therapy trial for advanced heart failure.

Cytori is starts to sell some of its StemSource® cell banking systems for collecting, processing, and storing adipose-derived cells. The company received its first order from Asian distributor Green Hospital Supply. The cell bank is to be set up in Japan. Despite all the continued skepticism about the value of cell banking to the donor-potential-patient, cell banking businesses are popping up prolifically around the world on the promise of short-term revenue to fund long-term R&D while providing a service some believe to be of irreplaceable value.

Although technically not a company-related cell therapy story, one of the more interesting developments this week was the announcement out of Charite in Berlin that they have a patient who is AIDS-free following a unique stem cell transplant. The patient developed leukemia which the docs were going to treat with a stem cell transplant. But rather than do the routine transplant by finding a match and treating just his leukemia they dared to be a little different. They went looking for a donor that matched and also had a CCR5-gene mutation (a naturally-occurring mutation which makes life difficult for the HIV virus according to some studies). The Berlin team found 80 compatible bone marrow donors for their patient, an unusually high sum, two of whom had the CCR5-Gene mutation. One of these they used with surprising and heartening results. What I like about this story almost as much as the happy ending and exciting discovery is how the doctors are trying to control the hype around this single-patient data and early results. It's not likely that this kind of transplant is a future treatmtent (for reasons related to supply, demand, and other commercial realities) but perhaps there is a kernal of a cell therapy product here somewhere that someone as innovative as these doctors will develop in time.

Ok I'm late on the draw here but it just came to my attention that in August NovaRx initiated a pivotal phase III in lung cancer patients. Watch on this blog in the coming days for more on the perception-vs-reality gap related to the number of industry-sponsored cell and gene therapy trials currently active worldwide.

Introgen may disappear while on the brink of success. Despite have an MAA being reviewed in the EU for their gene therapy for head and neck cancer, their BLA submission for the same therapy was rejected by the FDA as incomplete (currently under appeal). The company is now "exploring options" after a troubled Q3.

While not for its cell therapy program, Athersys announced it had achieved a clinical development milestone in its Bristol-Myers Squibb alliance. The amount of the milestone payment has not yet been revealed but any revenue is good revenue to offset the cell therapy development burn.

Bucking recent trends, StemCells Inc. (STEM) announced it will raise $20M in a stock sale to select institutional investors. Good news for any company in these times. Even better news for the cell therapy sector!

Finally, in the most over-reported news of the day, Pfizer opens up a regenerative medicine shop. I agree this is big news. This is not a minor investment; it's not throwing some money at a portfolio of biotechs in hopes that something will stick; it's not using stem cells as tools for drug testing or discovery. This is evidence of a belief by those in the halls of some of the tallest towers in life sciences, that cell therapies are going to change the way we treat people. Here are a couple quotes:

Pfizer Regenerative Medicine represents a great opportunity to focus world-class research in a field that holds considerable promise for biomedical science and for the treatment of many debilitating conditions such as diabetes and neurodegenerative disorders.” said Rod MacKenzie, PhD, head of Worldwide Research at Pfizer.

The unit will be led by Chief Scientific Officer, Ruth McKernan, PhD, who said “I’m very excited to lead this new research unit. While there is still a lot to understand about how stem cells can be used therapeutically, we believe it is one of the most promising areas of scientific research.”

Ok, one more. Corey Goodman, PhD, President of Pfizer’s Biotherapeutics and Bioinnovation Center, said, “The formation of this new unit represents another key step forward in Pfizer’s commitment to be at the forefront of new approaches in biotherapeutics and bioinnovation and to expand our research efforts and expertise into emerging areas of biomedical science, like regenerative medicine, that have great potential for human health.”

Apparently the unit will operate independently (in the fashion of smaller biotechs) with about 70 researchers when fully staffed - in both Cambridge, UK and Cambridge, Mass - and work collaboratively with "leading academic, biotech and pharmaceutical partners around the world.


That's the cell therapy industry as I saw it this week. Happy weekend.

Tuesday, November 11, 2008

one-question survey


I am trying to pull together some data on "perceptions" regarding the cell therapy industry and ask that you complete my one-question survey by clicking here: http://tinyurl.com/62brra

Thank a veteran today

We may not agree on everything...

Veterans Day

We may not even agree on the need for war.
But we can agree that our world would not be what it is today without brave service men and women willing to fight oppression and protect freedoms.


Thank a veteran today.

Friday, November 7, 2008

Cell Therapy HiLites 2008-11-07

I'm not going to turn this blog into a news service by any means, particularly since I wouldn't want to compete with what I already created in Cell Therapy News - still the single best news source for this sector. I'm going to aim, however, to put out a brief weekly email highlighting what I believe to be some of the key content I noticed during the week (news or otherwise).

For what it's worth, here's what I noted this week.

Athersys, Progenitor Cell Therapy, NeuroNova, Stem Cell Sciences, and Amorcyte featured in GEN article on regenerative medicines. GEN Oct 15 2008 (Vol. 28, No. 18)

Onyvax publishes data supporting the notion that the intersection of biomarkers, personalized medicine, and cell therapy is just around the corner and it will turn medicine on its head. They believe they have identified a
biomarker profile that could be predictive of response to cancer vaccines.

Science Direct's
Top25 Hottest Articles for 2008 Q2 have a very high ratio stem cell-related publications.

It was a big week for BioLife Solutions with several new adoptions of their preservation and storage media as well as the filing of their second MasterFile with the FDA.

On the economic front, the Genzyme-Osiris deal (worth a potential $1.4B) was obviously great news for a sector so keen to see signs that investors aren't shunning the field but on the other hand is something of a confirmation that companies will have to get creative to survive what Burrill projects will be a lengthy dry-spell in terms of biotech investment.

Invitrogen & Applied Biosystems resurrect the Life Technologies brand as the new name for their merged entity.

Sartorius Stedim relieves us of the confusion of having two Wave Biotech's out there with single use bioreactor systems (SUBs) by purchasing the European Wave Biotech AG. (full story)

The FDA takes a new approach to the annual HCT/P Establishment Registration Updates. (link)

Finally, if you haven't checked out www.biobusiness.tv yet you really must. Jean-Loup Romet-Lemonne has created a valuable biotech business video content channel that promises to focus a great deal on cell-based therapies because of Jean-Loup's experience in and passion for cell therapies.

Until next week...

Wednesday, October 22, 2008

Don't yet feel the pain of ignoring social media? Just wait a minute...




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 13 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. In the end, the only thing that convinced them of the need for a website was that it would likely be a good recruiting tool because it would make them seem hip to all the kids coming out of school. They finally also acquiesced to email only because it was seen to be an easy way to exchange documents with fellow attorneys.

How quickly they were proven wrong.

On a similar front, not that long ago many companies were caught without the domain names they would later need - or wish they had - related to their company names, product names, IP, etc. The most coveted .com domain name turf went to those with early foresight to be proactive and/or those with deep enough pockets to buy back the domains they wanted. Many companies wished they had not ignored the importance of domain names to their overall IP and business strategy.

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, you'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.

For those thinking this is just for tech companies or small companies (with small budgets that can't afford traditional media), you're also wrong. Pieter Kim has put together an enlightening list of examples of big companies now using social media: (http://www.beingpeterkim.com/2008/09/ive-been-thinki.html).

Let's start with some basics. I'll define "social media" as the 'interactive' side of the internet. So not the 'passive' sites where there is a one-way feed on information but rather Web 2.0 - those sites that have the capacity for readers to provide content, interact with the author/each other, and/or post comments or feedback. I'm talking about blog, podcasts, online communities, discussion boards, Wikis, and social networking sites.

This year Bioinformatics LLC announced the results of a survey of 1,500+ scientists in the life sciences primarily from North America (47%), Europe (36%), and Asia (11%) (http://lifesciencesocialmedia.com). Among their top 10 findings were the following:

1. Scientists interact with various forms of social media intermittently throughout the day
  • 27-32% spending1-2 hrs/day on blogs, content aggregators/portals, discussion boards, social networking sites, audiocasts, and/or wikis).
2. Social media provide scientists with a fresh perspective in their decision-making process
  • 30% said it has helped them "discover more options to solve a problem
  • 24% "faster access to news, research, and trend information"
3. Social media appeals to the most fundamental values of science — communicating, contributing and collaborating.
  • ~50% of respondents said blogs, discussion boards, and/or social networking sites "facilitate the sharing of ideas with colleagues and/or the scientific community"
  • ~30+% said blogs, content aggregators, podcasts, and wikis allowed them to "make a more educated decisions about purchasing new products and/or technologies"
  • ~40% said content aggregators, podcasts, and wikis made "it easier to research new products and/or technologies"
4. Tried-and-true discussion boards still dominate the social media landscape (50%) but online communities/social media sites were used by 30% of respondents in their "research and/or professional activities". Only 23% said they did not use any social media of any kind in their "research and/or professional activities".

5. Over half of respondents said they participate or "visit but don't participate" in online communities for their "research and/or professional activities.

6. Scientists agree that social media has influenced their purchasing decisions — but not the purchasing process.

7. Scientists want content that helps them better do their jobs. The top objection to using online communities was that "it would take too much time to maintain". Yet among users of social media, one of the top benefits cited was that it helped them do their jobs better.


There is no question the internet is now considered a primary resource of passive information access. Web 2.0's social media is all about making the internet the place to exchange information. Just as the best part of a lecture is often the post-talk Q&A or informal networking, web 2.0 is about online discussions. Users/participants of social media know that to maximize the value of these tools, one must share. The biblical maxim, "Give and you shall receive" applies here. You get out the same kind of value you put in. It's a discussion. It only works if both sides contribute. As with any form of networking, it always comes down to exchanging value. If you're only about self-promotion and not sharing something of value, you will be ignored and the benefits of social media will elude you.

Increasingly your constituents (customers, collaborators, investors, patients, employees, etc) expect to see you making a contribution to the online community. For many, being a good corporate citizen involves contributing value and exercising transparency and accountability. Increasingly they will select to do business with those companies they see and interact with online.

J&J and other pharmas now have blogs used to inform and interact with their constituents - media, patients, investors, etc. Many now have patient-oriented and/or disease-specific websites loaded with social media features. Even the NIH is using YouTube for clinical trial patient recruitment.

It is not enough now to build a website. For example, I spoke with a potential client just this week moaning that despite having a site for over a year it simply isn't showing up on search engines. You have to position your online content so people find it, have content that is useful to people vs simple self-promotion, and find ways to engage people: make them participate and keep them coming back.

Think beyond the website. Think professional society sites (e.g., ISCT, blogs). Think content aggregators (e.g., Cell Therapy News, BioSpace). Think business utilization of social media tools (e.g., LinkedIn, Facebook). Think blogging - corporate blog, employee blogging, following and commenting on blogs, and microblogging (e.g, Twitter). Think online discussion boards - monitoring for and posting content related to your business. Think using online tools that enhance search engine optimization and profile (e.g, Wikis). Think online audio-video content (e.g., podcasts, YouTube, Flickr) for education, training, profile, patient-recruitment, and general profile among media, investors, employees/recruits, etc.

And just in case you're still not convinced that your company must have a social media strategy, consider that your employees and customers are already there and likely dropping your company name. Here's a couple examples. I read people all the time on Twitter who regularly slag their job talking about things their boss would likely cringe at knowing were not just shared with a few friends but with all of Cyberspace in a searchable, eternally-archived way. If anyone is looking to work at their firms, a few online searches will bring up these threads. Another story I heard first-hand recently was of a young employee who was simply posting events of his day online including the troubles they were encountering with the technology s/he was working on. Information of the kind that would have been very informative to the competitor and not so comforting to the customers!

One simple application with enormous and immediate potential ROI is leveraging social media for sales and customer support. Online discussion groups or user groups are the easiest way to support customers and provide an information database for CSRs and product development teams. Building and mining "contacts" on social media sites is the modern-day version of the rolodex for sales teams. Contact lists easily convert to lead sheets, newsletter subscribers, and customers. Active participants in topical groups on social media (e.g., the Cell Therapy Industry group on LinkedIn) can be used to identify thought leaders, champions, detractors, etc. Furthermore they can be used as idea generators, beta thought-testing, focus groups, recruitment tools, etc.

Finally, hearkening back to my lawfirm days...maybe you just decide to use social media to recruit the new, smart kids. Just remember, its a little different world online so make sure your participation is genuine, transparent, and of value. With recruitment becoming increasingly competitive, leveraging social media may well be the only way to find and attract top talent.

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...