Cell Therapy Industry HiLites 2009-01-23

I hope to see many of you next week at the Phacilitate Cell & Gene Therapy Forum in Washington, DC. I suspect we are all today much more keenly anticipating Jane Lebkowski's talk (Geron) than we were last week.

I will be giving a short summary of the cell therapy sector in a lunchtime symposium on Tuesday sponsored by Progenitor Cell Therapy. I'll also be there with the founders of BioBusiness.TV interviewing executives, delegates, and doing some face time of my own in front of the camera all in support of an effort to launch a cell therapy channel on the new up-and-coming biotech business video site. We're looking for a sponsor for this cell therapy content - if you're interested, let me know - I think it's great value!

As a result of my being away all next week, I will more than likely skip the Industry HiLites posting next Friday. I'll try not to lose any news in the interim between reports.

While it's tempting to lead off with the news of Suzanne Somers' appearance this week for NeoStem (NYSE Alternext US: NBS) on CNBC's "Conversations with Michael Eisner", I suppose rightly so the big news of the week (year? decade?) is clearly Geron's news of FDA approval for its embryonic stem cell-derived therapy (GRNOPC1) for thoracic spinal cord injury patients. I dedicated a brief post last night to the news I helped break based on an early leak by the Morris Daily Herald. I also chewed up a fair amount of Twitter bandwidth this morning doing 'live tweeting' during Geron's webcast. (see http://twitter.com/celltherapy). At midnight (EST) Thursday night, Geron updated their website with everything they're willing to share about their product, trial, manufacturing, testing, etc. It's actually quite informative >> click here.

Earlier in the week in-PharmaTechnologist.com discussed Lonza's implentation of a plan to spend"$26m to expand capacity at its facility in Walkersville, Maryland, "as regulatory progress made with Osiris’ Prochymal looks set to kick start the era of cell therapies". This will add capacity by Q1 2010 to meet anticipated demand by its customer Osiris for production of Prochymal should it be approved.

On the fundraising front, Belgian biotech Cardio3 BioSciences has rounded up €13.7 million from venture groups and grants. Cardio3 raised €7.2 million in a Series B and an additional €6.5 million in cash advances from the Walloon Region General Directorate for Economy, Employment & Research. Much of the new money will be used to fund a 240-patient clinical trial of C-Cure, its lead program. C-Cure is designed to allow the differentiation of a patient's own cells into 'cardiopoietic' cells which grow into new heart cells and repair heart muscle.

Additionally, International Stem Cell Corporation (OTCBB:ISCO) has received the second $1 million tranche of an anticipated private equity financing of up to $5 million to be funded over the next several months. As we speculated might be the case a few weeks ago, the investors were the same as the last round: X-Master, A. Semechkin, and R. Semechkin. According to a recent SEC filing X-Master is a New Hampshire corporation which provides software consulting and computer hardware services and which holds real estate and other assets and investments. The SEC filing also reported that the source of funds for the acquisition of the shares in the previous rounds was primarily from the proceeds of loans made to X-Master by Hartcom Impex, Ltd., a British Virgin Islands corporation and from the working capital of X-Master. Not your garden-variety financing by any means.

After much delay, ReNeuron has received permission from UK authorities to launch a phase I trial for stroke patients using their neural stem cell product ReN001. The ReN001 cells will be administered by direct injection into the affected region of the brain in a straightforward surgical procedure.

The Company submitted an application to the US FDA some time ago to commence a clinical trial in the US with its ReN001 stroke therapy. This application has not been rejected by the FDA, but remains on clinical hold. The Company intends to continue discussions with the FDA in due course concerning this application and potential amendments thereto. The company may need to identify a safe and effective cell labeling technology to track these cells before it wins approval in at least some other jurisdictions outside the UK.

Financially, the Company intends to raise further funding for its programes over the course of this year, and its convertible loan facility from certain existing investors will provide cash resources sufficient to finance the Company's operations into the third quarter of this year. (see articles in the Financial Times and NatureNews for more details).

Angel Biotechnology Holdings plc (AIM: ABH) issued a press release congratulating ReNeuron on receiving this approval because Angel is, of course, ReNeuron's manufacturing partner.

BioBusiness.tv has posted a great interview of Osiris CEO, Randy Mills by Bill Kridel of Ferghana Partners Group. Randy dares to use the word "blockbuster" in conjunction with his expectations for Prochymal. He also reports that Osteocel has now treated 30,000 patients since its launch in July 2005.

Nuvasis, Inc (NASDAQ: NUVA) is projecting $28M in Osteocell sales in 2009. While that's only 8% of its overall revenue outlook of $350M, it's not insignificant. Add that to over 250,000 patients treated to-date by Appligraf (Organogenesis) for total revenue in 2008 alone of ~$70M, ~13,000 patients to-date for Carticel (Genzyme), and the fact Advanced Biohealing's facility is able to produce 250,000 units of Dermagraft per year....and we just might have the beginnings of a viable sector here.

Mesoblast (ASX:MSB) received approval from the Australian regulators to proceed with a randomized, placebo-controlled phase II trial of their allogeneic stem cell product, RepliCart, for knee osteoarthritis after acute traumatic injury. StemCellDigest immediately commented on the potential patent questions vis-a-vis Osiris.

TAP launched its automated 'CellCelector' at SelectBio's Stem Cells World Congress. The device consists of an inverted Olympus microscope, robotic arm and liquid handling station integrated with image acquisition and analysis software. The system allows researchers to set parameters for cell or colony types they want (including size, proximity to other colonies or roundness). The picking tool on the robotic arm gently picks and dispenses cells into a microplate well in just 30 seconds. CellCelector can fit into any standard laminar flow hood and can be fitted with an autoclave compatible metal tool for scraping adherent cells or a disposable glass capillary for picking single cells.

StemCellDigest.net summarized The Money Raising Sagas Of Advanced Cell Technology Inc.

Amidst circulating rumors and clear evidence of financial distress, Opexa Therapeutics, Inc. (NASDAQ:OPXA) - developing patient-specific cellular therapies for the treatment of autoimmune diseases such as multiple sclerosis (MS) and diabetes - provided an update on corporate activities.

Looks like a PerkinElmer has a strategy congealing around its ViaCord/ViaCell acquisition. After announcing a collaboration with MD Anderson last week, this week they announced that its ViaCord Research Institute, which focuses on supporting science, technology and medical treatments using cord blood stem cells, will support the University of Massachusetts Medical School (UMMS) in its research efforts into the potential use of umbilical cord blood-derived stem cells in treating type 1 diabetes.

According to a recent report, while more than 10,000 Australian parents have paid between $3000 and $5700 to have their child's cord blood collected, frozen and stored in the seven years since private collection companies began operating in Australia, no samples have been used for medical treatment.

This one is more the news behind the news. Novartis has recently posted a position for a Regenerative Medicine Ophthalmology Research Investigator PhD/MD. The description? "The candidate will lead a research team dedicated to developing regenerative medicine therapeutics for ophthalmic disease. The team will collaborate with other technology groups within the Novartis Institutes for Biomedical Research (NIBR) to identify novel chemical and biologic modulators of adult stem cells, validate the therapeutic potential of these discoveries, and develop these assets into therapies targeting blinding eye diseases with a high unmet need." So...while it would appear they're stuck on finding non-cell therapy regenerative compounds, they're playing with stem cells. That's a good thing.

With all the other noise around Bioheart (NasdaqCM: BHRT) last week, I missed some important potential good news. Bioheart, Inc. announced last wek that they have filed with appropriate agencies in Switzerland for reimbursement approval for its MyoCell(r) myogenic cell therapy for heart failure. The initial reimbursement application filings are targeting the sickest Class III and IV heart failure patients who have not responded well to drugs and do not qualify for a bi-ventricular pacemaker. Bioheart is applying for a reimbursement level matching exactly that charged to provide bi-ventricular pacemakers for this same patient population.

So how can companies like Bioheart file for 'reimbursement' before the therapy is approved? I asked this question of Eric Faulkner of RTI Health Solutions. He provided this answer. "This is not a filing for ‘full reimbursement’ but is a filing for reimbursement subsidy for their clinical trials in certain target markets. Usually these are competitive and not every thing gets funded, but if approved the manufacturer likely covers some costs (e.g., cells, catheters, etc.) and the hospital would be covered by the govt. (e.g., some of the inpatient facility and provider costs). This can be a way to defray costs of conducting the trial leading up to regulatory market clearance. CMS and some limited # of US commercial payers have similar provisions in areas of high unmet need." I learned something.

Linda Powers took advantage of all the hype and interest around Geron's news to remind the world through an article by John Sterling in Genetic Engineering News that her $140 million Toucan Capital venture capital fund "holds the largest number of stem cell, regenerative medicine, and living cell companies in the world [16 companies]". She also stated "not enough attention is being paid to the biomanufacture of stem cells and other living cells" like is done by her portfolio company Cognate Bioservices.

Lord knows looking at my stock portfolio, I'm no stock analyst - not even a good stock picker. With that in mind (i.e. this ain't no stock recommendation or even general negativity about Dendreon), below is a little analysis on Dendreon this week by David Gaffen on Wall Street Journal's MarketBeat blog that I share with you more for what it tells me about the trial, the data, and analysts' thinking than for what it might mean for the stock:

It’s been a rough week for Dendreon, a biotechnology stock with a fervent group of followers that wait with baited breath for every bit of news about its Provenge treatment for prostate cancer. The stock was hit hard earlier in the week after news that data from company’s ongoing trial for its treatment will be released in April, earlier than expected, because the trial has surpassed the threshold for number of deaths to incur a final analysis. The problem, as some Wall Street analysts see it, is that the company amended its trial late in the process to include sicker patients. The drug needed to show a 22% reduction in the risk of death to be successful, and it was at 20% earlier in the trial. “We firmly believe that the death events occurring after the May 2008 cutoff date consist of a greater percentage of patients that were enrolled later in the trial (i.e., sicker patients), which would make it even harder for any drug to offer interim analysis,” write analysts at Brean Murray Carret & Co. “Therefore, the 20% difference reported at interim is actually further from the 22% goal required at final analysis than it may seem at first glance.” Some are remaining optimistic, however, including David Miller, who writes on Minyanville.com that “long-suffering Dendreon bulls (like me) will be rooting for those extra 2 points of patient benefit.” This stock has been a volatile one — and if the 22% threshold is reached, expect wild action. Shares hit a 52-week low of $3.42 Tuesday.

Finally for you lab-heads and quality-geeks out there... ISCT is offering a webinar February 18, 2009, entitled (Part 1): Validation and Qualification of Equipment and Reagents.

And that wraps up a momentous week for cell therapy. I love the smell of stem cells in the morning...

Safe travels.

The Perfect M&A Storm is Brewing.

I'm not going to write only about the financial side of cell therapy sector but I'm on a roll here so I'll continue on the theme for at least on more post.

I've been privy to a lot of conversations recently in which M&A is on the tip of everyone's tongue. I'm likely not going out on too much of a limb - and certainly won't be the only one on it - when I make the prediction that the next 12-24 months is going to see a spike in M&A activity at all levels in the cell therapy industry.

I don't think I need a hard data set to convince you that we've seen an increase in M&A activity over the past 6 months.

This I know:

• There is a fair amount of M&A activity right now in the sector.
• The M&A pendulum has now swung back to the "sexy" side after falling out of favor for some time.
  
• A high percentage of companies in the sector are expressing interest in some level of merger or acquisition.
  
• There are a lot of M&A discussions currently being explored.

Certainly we're seeing this in the larger biotech sector with Roche's offer for Genentech, MacroGenics' acquisition of Raven Biotechnologies, Lonza's purchase of Amaxa Biosystems, and Invitrogen's merger with Applied Biosystems as prime examples just in the past few weeks alone.

There is a perfect storm of factors brewing up the ideal climate for a spike in M&A. VCs are increasingly tightfisted and conservative about life sciences for now, capital markets are tough with predictions it won't get better for some time (see today's IN VIVO blog), and - to round out the trifecta - the sector's maturation translates into increasingly larger burn rates for a higher number of cell therapy companies in later stage trials.

I'm not in the business of spilling confidential information or spreading rumor so any companies mentioned below are used simply by way of example. Don't assume I know something you don't know about any of the companies referenced below.

So what might this potential M&A dustup look like?

It will certainly involve many smaller companies - think Onyvax, KeraCure, Aastrom, Memgen, ReNeuron, HemoGenix, Novocell, Opexa, IRX, BioLife Solutions, BioSafe, Cellerant, Eufets, Progenitor Cell Therapy, Angel Biotech, Epiontis, ISCO, Lifeblood Biological Services, Stem Cell Sciences, Thermogenesis, TiGenix - some of which will look to M&A to solve their fundraising needs and others will be M&A targets because of their revenue stream.

Larger companies are not likely to be immune from this swing of the M&A pendulum - say (for example!) Dendreon, Neurotech or even - might it be possible - Osiris? The newly improved, profitable, and growing Organogenesis might be a target for its revenue and Stem Cells Inc has been none-too-shy about its M&A intentions for some time. It may involve some bigger companies in the sector - say like Genzyme or Caridian BCT (formerly GambroBCT) which we know is almost certainly being positioned for a resale by the private group that bought it to maximize its value for resale.

What is not clear is who might be the buyers. Potential companies that pop to mind include BD , Celgene, and J&J. Might Amgen make a play? What about Shire, Schering, or any of a number of other pharma looking to diversify their sagging pipelines and bolster their lackluster innovation initiatives. There will likely be other players external to the field who use M&A as an entree into the sector - think Reliance Life Sciences

We know Hospira is making a play in the cell therapy space with several investments they won't yet disclose. They hiring key and experienced executives (e.g., K. Gunter from ViaCell some time ago and recent D. Perritt from J&J). It would appear they are positioning to make a significant play in the market and it would not be surprising to see it in the services side of the sector.

It is likely to be an interesting year or two for cell therapy. On the other side of it, the industry will likely look much different, be more robust, held by a much wider diversity of parent companies, investors, and shareholders, and likely somewhat more integrated with other biotech/pharma.

So...make sure your boat is tied up, get a good window seat (if you're a watcher) or a kite sail (if you're want to participate), and let's have fun. Let's make sure smart deals get done that get good technologies in the hands of companies that will make them work - as therapies, as the base of sold companies, and rewarding to investors with the cojones & foresight to put cell therapies in their portfolio.