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Friday, January 23, 2009

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 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 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. 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. 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 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.

Thursday, January 22, 2009

Geron obtains FDA approval for phase I human embryonic stem cell study

According to reports filed tonight, the FDA has approved Geron's clinical trial of embryonic stem cells for spinal cord injury.

Why the Morris Daily Herald gets the scoop on this Associate Press story is a mystery to me but according to an article filed this evening in the Morris Daily Herald, picking up the feed from Associated Press science writer Malcolm Ritter, Geron (Nasdaq: GERN) received approval to proceed with its trial "this week. "
"'The company gained federal permission this week to inject eight to 10 patients with cells derived from embryonic cells,' said Dr. Thomas Okarma, president and CEO of Geron Corp."
The timing of this is ludicrously delicious. The FDA just happened to wait to approve the trial until Obama is sworn into office? And/or did Geron just happen to wait to release the news until the new Administration came in? I can't believe there was political pressure or influence but the timing of this is admittedly freaky.

When I started writing this the news had not yet come from Geron. Then their site went down. Now it's up on Geron's home page. It's official!

There are people who question Geron's decision to go to the clinic in a spinal cord injury model and others who have have critiqued Geron's business model or corporate style but there is no question that today is not only Geron's day but - if this is true - it is a red letter day in the inevitable progress of cell therapy, stem cell research, and patients with spinal cord injury.

"'It's a milestone and it's a breakthrough for the field'.. said Ed Baetge, chief scientific officer of Novocell Inc."

This marks a new day for embryonic stem cell research and the imminent lifting of the restrictions on federal financing of embryonic research will be icing on this cake.

It will be most interesting to see now the effect on the fortunes of other stem cell companies - embryonic and adult - cell therapy companies generally and, in particular, the stocks of those which are publicly traded.


Here is a copy of the full article from the Morris Daily Herald:

Last modified: Thursday, January 22, 2009 6:03 PM CST

US approves 1st stem cell study for spinal injury

AP Science Writer

NEW YORK (AP) — A U.S. biotech company says it plans to start this summer the world's first study of a treatment based on human embryonic stem cells — a long-awaited project aimed at spinal cord injury.

The company gained federal permission this week to inject eight to 10 patients with cells derived from embryonic cells, said Dr. Thomas Okarma, president and CEO of Geron Corp. of Menlo Park, Calif.

The patients will be paraplegics, who can use their arms but can't walk. They will receive a single injection within two weeks of their injury.

The study is aimed at testing the safety of the procedure, but doctors will also look for signs of improvement like return of sensation or movement in the legs, Okarma said.

Whatever its outcome, the study will mark a new chapter in the contentious history of embryonic stem cell research in the United States — a field where debate spilled out of the laboratory long ago and into national politics.

While some overseas doctors claim to use human embryonic stem cells in their clinics, stem cell experts said they knew of no previous human studies that use such cells.

"It's a milestone and it's a breakthrough for the field" because Geron passed the safety hurdles for getting federal clearance to launch the study, said Ed Baetge, chief scientific officer of Novocell Inc. His company hopes to begin a similar human study for treating diabetes in a few years.

In addition, said spinal cord injury researcher Dr. Wise Young of Rutgers University, "a lot of hope of the spinal cord injury community is riding on this trial."

Embryonic stem cells can develop into any cell of the body, and scientists have long hoped to harness them for creating replacement tissues to treat a variety of diseases. But research has been controversial because embryos must be destroyed to obtain them.

President Barack Obama has promised to relax the Bush administration's restrictions on federal financing for such research. But Obama's ascent to the White House had nothing to do with the U.S. Food and Drug Administration's granting permission for the new study, Okarma said in a telephone interview Thursday.

In fact, the company says, the project involves stem cells that were eligible for federal funding under Bush, although no federal money was used to develop the experimental treatment or to pay for the human study.

Other human cells, called adult stem cells, have been tested before in people to treat heart problems, for example.

In the Geron study, the injections will be made in the spine at the site of damage. The work will be done in four to seven medical centers around the country, Okarma said.

Animal studies suggest that once injected, the cells will mature and repair what is essentially a lack of insulation around damaged nerves, and also pump out substances that nerves need to function and grow.

Apart from assessing safety, investigators will hope to see some signs of improvement in the patient, Okarma said. The idea is "not to make somebody ... get up and dance the next day," he said, but rather to provide some level of ability that can be improved by physical therapy.

Each patient will receive a low dose of anti-rejection drugs for about two months, because after that time the medications shouldn't be needed, Okarma said. The study will follow each patient for at least a year.

Okarma said he can't estimate how much such a therapy would cost if it proves effective, but that "this is not going to be a $500,000 price tag. It will be remarkably affordable ... in the context of the value it provides."

Evan Snyder, a stem cell researcher at the Burnham Institute for Medical Research in La Jolla, Calif., said scientists in the field will focus chiefly on the study's results about safety.

"The one hope that everybody has is that nothing bad happens," he said.

Geron Corp. has spent at least $100 million on human embryonic stem cell research. Founded in 1992, it does not have any therapies on the market.

However, the company is considered the world's leading embryonic stem cell developer thanks to its claims on several key stem cell technologies. Geron helped finance researchers at the University of Wisconsin who first isolated human embryonic stem cells in 1998. The company has retained exclusive rights on several of those cell types.

Friday, January 16, 2009

Cell Therapy Industry HiLites 2009-01-16

Ok, seriously, this week's HiLites blog almost killed me. Hope you find it useful. This was the week for cell therapy I thought last week would be. I'm going to try and recover now...

Dendreon's (NASDAQ: DNDN) Mitch Gold announced at this week's JP Morgan conference (click here to hear the presentation) that the IMPACT study of 500 men with terminal prostate cancer will offer a final answer by April on whether its experimental immune-boosting drug helps patients live longer. When the company went to the FDA with its BLA for PROVENGE in late 2006 and - despite the advisory committee's 13-4 vote recommending approval (March 2007) - the FDA voted against approving it at that point, it was this trial the FDA said they would prefer to see the data from before deciding. There is, as always, a much longer checkered history of ever-changing FDA reviewers, missed clinical endpoints, retrospective data analysis to find a patient subset for which the therapy worked, etc, etc all of which are partial drivers I'm sure for deciding to wait for the data from this second phase III study --- but it all comes down to this. In a mid-point preliminary review of the data, the endpoints were not met - but they were almost met. Mitch Gold is, of course, confident. As was the case in early 2007 there will be much speculation and the stock will be traded wildly and the cell therapy industry will wait with bated breath - though perhaps not quite as bated as 2 years ago when it seemed more rode on the decision than it does now.

The company has built its own manufacturing facility on (perhaps in due course only for) the US East Coast (New Jersey). They still own the worldwide rights to market the drug. They intend to go to the US market with a ~120-person sales force and plan to seek a commercialization partner for marketing outside the U.S.

In other late-stage cell therapy news, Osiris had a successful pre-BLA meeting with the FDA in which they reportedly reached agreement on the timing and content of their anticipated BLA submission. This will be the first US marketing application for a stem cell product making it much-anticipated, globally monitored, and precedent-setting. The entire industry has been waiting for this moment for several years (the last cell therapy approved being 10 years ago!) and will be watching this in much the same way as we were all watching Dendreon's BLA submission a couple year's ago.

Reported key results of the pre-BLA meeting were:
• FDA agreed with the proposed content and structure of the BLA for Prochymal for the treatment of GvHD.
• The primary endpoint of the Phase III trial that will be reviewed by FDA for approval was confirmed to be GvHD Complete Response (CR). A CR is complete remission of the disease.
• FDA indicated that the statistical analysis plan was appropriate.
• The overall safety database (Integrated Summary of Safety) of Prochymal is sufficient in scope for the indication. No additional clinical data beyond what was presented is anticipated to be necessary for the BLA.
• Agreement was reached on BLA requirements for Chemistry, Manufacturing and Controls (CMC) data.
• The FDA concurred with the proposed product stability and process validation plan for submission and provided guidance on how to present the data for ease of review.
• Agreement was reached on the timing and content of the BLA in rolling submission format. The rolling submission is an FDA provision available to drug candidates that have received Fast Track designation, which allows for completed sections of a BLA to be submitted on an ongoing basis. It can facilitate the process by allowing FDA to complete review of sections as soon as they are available. It is anticipated that the nonclinical sections, such as toxicology, will be the first submitted for review.
• The BLA will be in the electronic Common Technical Document (eCTD) format. The eCTD format facilitates the review of the BLA and allows parallel submission of the dossier in other territories.
• At the time of submission, Osiris will also submit the request for Priority Review of the Prochymal BLA. Investigational drugs with Fast Track designation are eligible for consideration for Priority Review, which provides for an accelerated six month application review by FDA

In other news not to be overshadowed by the news about the pre-BLA meeting, Osiris received FDA clearance to broaden the Prochymal Expanded Access Program (EAP) to patients of all ages suffering from life-threatening GvHD. In May of 2008, FDA approved the first Prochymal EAP for the treatment of pediatric GvHD patients. Congress and the FDA created the expanded access program to facilitate the availability of promising new drugs to desperately ill patients before general marketing begins.

If you're interested, click here for an interview of Osiris CEO, Randal Mills, done by this week.

Invitrogen launched a xenogeneic-free media for human embryonic stem cell culture.

After now at least 4 years of GE Healthcare's campaign to build a 9-figure cell therapy division, a rocky relationship with Thermogenesis, and the splashy acquisition of Wave Biotech, GE has made another entree into the sector with its announcement of a partnership with Cytori (NASDAQ: CYTX) to commercialize Cytori’s Celution® System in select European countries - exclusive for 18 mos in select countries. While GE is admittedly not lighting any fires with the pace in which they are building this division it is presumably (hopefully) strategic, synergistic, supportive of its overall business and...please let this be true...something Jeff Immelt will continue to wholeheartedly support despite the perpetual and perpetual re-orgs within the company.

Celprogen claims to have successfully de-differentiated adult somatic cells (primary skin fibroblast, keratinocytes, cardiac, liver and neuronal cells) to their embryonic pluripotent cells (iPCs) using a proprietary non-viral method involving their de-differentation media and matrix system. The iPCs were then reportedly differentiated into cardiac, neuronal and hepatic tissue with Celprogen’s Stem Cell Differentiation system.

The PerkinElmer ViaCord Research Institute and M. D. Anderson Cancer Center have agreed to extend collaboration on a study of cord blood stem expansion systems in adult transplantation. The study seeks to discover methods of treating more adults using cord blood stem cells using 'co-culture' expansion technology. Based on the results of this trial, ViaCord, PerkinElmer's cord blood banking business, and M.D. Anderson will determine the feasibility of using Unrestricted Somatic Stem Cells (USSCs) derived from cord blood in the co-culture of cord blood units for transplantation. USSCs, proprietary to ViaCord (patent pending #09/985,335), are a type of stem cell found in umbilical cord blood, which have the ability to differentiate into many cell types, including endothelial cells, fat, bone, cartilage and neuronal cells.

ERYtech Pharma, French biotech company developing a pipeline of products involving the encapsulation of therapeutic molecules inside red blood cells for a variety of orphan indications, has signed an agreement with Penn Jersey Region of American Red Cross for the "GMP" clinical batches production of ERYtech Pharma’s products in the United States.

I'll seque momentarily to the regretably inevitable 'bad' news ...

On the verge of filing a BLA, Isolagen (Amex: ILE) is forced to downsize its operations to minimize burn-rate and postpone bankruptcy while exploring its 'options'. On November 6, 2008, the Company disclosed in an SEC Form 10-Q that there existed substantial doubt about its ability to continue as a going concern, and that its ability to continue as a going concern was contingent, among other things, upon its ability to secure additional adequate financing or capital prior to January 15, 2009 under its current operating plan and condition. The company announced it was pursuing dual paths, including pursuing potential financing alternatives as well as continuing potential strategic partnership discussion. As of this week neither event has happened according to an SEC filing.

The Company has now deviated from its normal operating plan and is focusing its remaining cash resources on specific clinical programs, namely efforts to complete its Biologics License Application related to the Company’s Phase III Nasolabial Fold study, and continuing its IT-R-008 Phase II/III Acne Scar trial. All other clinical programs have been suspended in order to preserve the Company’s remaining cash resources. The company believes it can survive for awhile but not sure how long. Its largest debt is a $90M liability which can be called by one of the noteholders as early as November.

Arbios Systems, Inc. (OTC: ABOS) has filed for chapter 11 bankruptcy protection while they continue to seek bids for the sale of the Company and/or its assets - namely the SEPET™ Liver Assist Device (a blood purification therapy designed for use with a standard blood dialysis system). The company shuttered its operation in August to focus "day-to-day operations exclusively on obtaining financing or consummating a strategic transaction'. In October the company sold all its rights and interest in its bioartificial liver system, HepatAssist, to HepaLife Technologies, Inc. (OTCBB: HPLF). This was really the cell therapy part of the company. The HepatAssist™ Cell-Based Liver Support System - a hollow fiber cartridge housing living porcine liver cells - purportedly functions as a bioartificial liver that not only detoxifies the blood, but, uniquely, provides whole liver function - intended to treat acute and acute-on-chronic liver failure patients.

This is a buyers market and the reality is many potential investors or purchasers are not motivated to buy a going concern for $x if they can scoop up the assets and/or business at a significant discount under distress. Regretable for those companies that have created and nurtured good technologies but allowed their business to fail; good news for those with cash; perhaps also good news - say some - for a much-needed reorg of an industry with a much more disciplined commercial focus.

Last month we talked a little about Bioheart (Nasdaq:BHRT) and their IPO less than a year ago raising just under $6 million. Now the stem cell company Bioheart has alerted the SEC that it missed a scheduled $180,000 loan payment and is seeking new money to keep the company going.

The Miami Herald reports that the Sunrise, FL-based developer has warned investors that it faces some tough choices including potential layoffs, bankruptcy and closing the business. But, reports Jim Carroll from Fierce Biotech reports, "the company's CEO, Howard Leonhardt, says the lender--BlueCrest Venture Finance Master Fund--has agreed to provide an extra 90 days for the company to make the payment. Bioheart, he adds, had to deal with an unexpected delay in receiving a $2.5 million loan and he insisted that the company is not in danger of closing."

According to Leonhardt, ''The last 24 months in a row we've paid on time and it was an unforeseen event. We're not thinking of bankruptcy at all; it's not even close to consideration -- maybe laying off employees.'' But all is not as serene as a pic of the debonair Leonhardt, accompanying the Herald story, would seem to suggest. A group of board directors and key executives have departed the company in the last two weeks, including CFO William Kline.

Nonetheless, there is reason to be optimistic despite the alarm bells. Bioheart - which is not just a one-trick cell therapy pony - recently announced the approval or release of four new products. In October, it launched in the United States and Europe a heart failure monitor that patients can use at home. Several major sales contracts are pending and the company has set a $20 million goal for 2009, Leonhardt said. It has also won FDA approval for the Bioheart Monebo CardioBelt, which heart patients wear around their waist, as well as commercial approval for two other technologies in Europe. Also, while I still have unanswered questions about who is supplying who with what, they also recently signed a cell supply agreement with Life Technologies to sell myoblast cell-based research products to life science researchers. By March, Leonhardt said, Bioheart should be on sound footing cash-wise and would try to fund operations solely with revenue and profits. Until then, however, ''we have to bring some more capital in now to bridge the gap,'' Leonhardt said.

On now to more optimistic fronts...

In a recent interview, CIRM President, Alan Trounson, said he (CIRM) is lobbying for the creation of an 'investment' fund - sponsored by government and health insurers - to drive commercialization of early regenerative medicines. He fears that without such a 'new paradigm', the lack of funding on the scale typically provided by the pharmaceutical industry, could well be a roadblock in regenerative medicines being brought to market. Trounson predicts within 10 years we'll have new drugs based on stem cell discoveries and clinical products being tested for spinal repair, retinal repair, cardiac function repair, multiple sclerosis, etc.

BioE recieved FDA 510(K) clearance for its cord blood processing system. PrepaCyte-CB is available immediately to public and private banks separating potentially therapeutic cells and stem cells from cord blood.

Pluristem Therapeutics (NasdaqCM: PSTI; DAX: PJT) filed an IND with the FDA to initiate a two-center Phase I clinical trial in the U.S. utilizing PLX-PAD - placental adherent stromal cells (ASCs) expanded in the Company's proprietary PluriX TM 3D bioreactor - for critical limb ischemia. The company filed a IMPD in Europe for a similar trial there.

The Swedish investment company Karolinska Development AB has made what it calls a "significant investment" in EvoStem Finland Oy - a company that develops stem cell and other tissue engineering based treatments for veterinary use (and intends to go human at some point). In 2007 EvoStem Finland Oy introduced to the Scandinavian market its first product TendoStem(R) - a stem cell based treatment service for tendon and ligament injuries in horses.

Kiadis Pharma has recently come out with a bunch of news (some of which I'm a little late reporting). Most recently they Dr. Maarten Egeler, MD PhD as Chief Medical Officer and in December they reported the appointment of Mark Skaletsky to its Supervisory Board and, most significantly, announced they have received two orphan drug designations for Reviroc™ from the FDA - one for diffuse large B-cell lymphoma and the other one for the treatment of follicular lymphoma. Reviroc™ is under development for the elimination of cancer cells from an autologous graft in bone marrow transplantations for end-stage blood cancer patients.

Skaletscky, for those of you who don't know him, is very well credentialed. He served as president and CEO of GelTex Pharmaceuticals, Inc. (acquired by Genzyme for more than $1 billion in December 2000), was chairman and CEO of Enzytech, Inc., and president and Chief Operating Officer of Biogen, Inc. He is also a former chairman of the board of directors of the Biotechnology Industry Organization (BIO). Currently he is chairman, CEO and president of Fenway Pharmaceuticals, Inc.

Let me digress momentarily with a little history and speculation...

In 2001, Theratechnologies (TSX:TH) reorganized its business activities through the transfer of its ex vivo photodynamic cell therapy program to its newly created subsidiary, Celmed BioSciences, the acquisition of NeuroGeneration and NeuroGenomics, two California corporations which were developing technologies for the treatment of neurodegenerative diseases, and investments totaling $60 million by Societe generale de financement du Quebec (SGF) and the Solidarity Fund QFL. Michel F. Levesque, founder of NeuroGeneration became Vice President, Medical Affairs with Celmed BioSciences.

In 2006 Kiadis B.V and Celmed BioSciences Inc. merged the two oncology-focused companies. At that time, CelMed Biosciences was a wholly-owned subsidiary of Theratechnoloies (another Montreal company). The new combined company was named Kiadis Pharma B.V. Investors include Alta Partners, LSP, and DFJ Esprit. At or around that time, Levesque took back NeurGeneration which he is now attempting to re-launch from his practice in Hollywood, CA.

Kiadis still describes itself as a "Dutch-Canadian enterprise" which is "endorsed by our investors, academic partners and a cross-Atlantic management team". There is much cross-pollination between Montreal and Amerstdam. For instance, Pierre Caudrelier, was until this week, CMO at Theratechnologies, was previously CMO at Kiadis Pharm, VP of Clinical Research at Celmed, and VP of Clinical Research and General Manager the the Canadian subsidiary of IDM (another company that had cell therapy products with US-EU connections). Research collaborations also exist with Italy, Germany and the US (including NIH's John Barrett).

Here comes the complete and utter speculation. On the one hand we have an unnamed fairly large multi-national company (not GE) looking for acquisitions in the cell therapy space. Links have been established with personnel at Theratechnologies - a company which I believe no longer has any cell-based therapy research. On the other hand, totally unrelated to this, I am led to believe that Kiadis is looking for a partner with money. Other than some personnel and history, there would not appear to be anything still linking Theratechnologies and Kiadis. However, just to make things a little curious Theratechnologies announced this week that Dr. Pierre Caudrelier was leaving his position as chief medical officer to pursue other career opportunities. Despite the complete lack of any evidence other than this tenuous string of bits and bytes, something tickles my senses here. I'm betting there is more to this puzzle than I have yet been able to put together (or am willing to say out loud at this point). Stay tuned.

On the regulatory front...

FDA CBER has announced their Cellular, Tissue and Gene Therapies Advisory Committee 2009 meeting schedule: May 14-15 and November 5-6

For those of you using genetically engineered pigs for to grow cells, tissues, or organs that can be transplanted into humans with a reduced risk of immune rejection, you will know the FDA this week issued its Final Guidance on Regulation of Genetically Engineered Animals. For the rest of you, I can't think of better night time reading...

The FDA issued a Draft Guidance for Industry entitled: "Current Good Tissue Practice (CGTP) and Additional Requirements for Manufacturers of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps)" [pdf version]. Although you can comment on any guidance at any time (see 21 CFR 10.115(g)(5)), to ensure that the agency considers your comment on this draft guidance before it begins work on the final version of the guidance, submit written or electronic comments on the draft guidance by April 16, 2009.

Let me mop up with a few final lighter entries.

Rurther to my blog on the subject back in October, BioTechniques just released interesting results on a survey of the extent to which scientists are using social media tools and which one for what. See more details in the comments section of my original blog post of social media use.

If you're planning to be at the Phacilitate Cell & Gene Therapy Forum in Washington, D.C. January 26-28 (and you should plan to be there if you have any interest in this field), here's a couple things to watch for:

- DCi Biotech consultant, Dawn Driscoll is moderating a lunchtime session on Tuesday the 27th hosted by Progenitor Cell Therapy in which yours truly, Robert Preti, and RTI Health Solutions, Eric Faulkner will be speaking. The session is entitled"Select Issues in Commercializing Cell Therapies" and will include a talk by Faulkner on positioning cell therapies for reimbursement.
For more details, click here.

- I will be there with the founders of BioBusiness.TV who will be there to capture some interviews from cell therapy industry executives for a cell therapy channel we are opening for the site.

- Anyone interested in potentially incorporating in vivo cell tracking/imaging in their cell therapy clinical trial, come find me. more thing just for kicks. For a chuckle and then a shake of the head in dismay check out the Niche blog on ESC-derivative facials! The hype is really getting out of control.


That's it. That's all I've got for you. That, my friends, is how the cell therapy industry hit me me this week.


p.s. Quick question: Do you mind/like this rather rambling format or should I get more organized somehow or another? Comment below...

Friday, January 9, 2009

Cell Therapy Industry HiLites 2009-01-09

Ok so there was no deluge of cell therapy news this week despite my fears (hopes?). The only flood was the river (nee creek) behind my house. Wow, water can rise fast! Anyway...that's not cell therapy, THIS is cell therapy:

Osiris Therapeutics (NASDAQ:OSIR) received a $5 million milestone payment for reaching the first production threshold of the Osteocel supply agreement with NuVasive. In July of 2008, Osiris announced the sale of the Osteocel business to NuVasive for an initial payment of $35 million, followed by up to $50 million in additional milestones. Osiris expects to achieve the remaining $45 million in milestone payments from NuVasive, including $17.5 million for further delivery of product, $12.5 million upon transfer of certain manufacturing assets, and a $15 million payment upon NuVasive’s achievement of $35 million in cumulative Osteocel sales.

After announcing last week that they had raised $1M of an anticipated $5M round of financing, International Stem Cell Corporation (OTCBB:ISCO) issued a press release to 'discuss' the recent funding. The additional funds are to be raised in four $1M tranches in February, March, June and September.

They need the additional funds to meet their goals for 2009, namely
(1) eliminating $1 million of outstanding secured debt;
(2) supporting first quarter pre-clinical trials; and
(3) providing marketing and expansion capital to position the Company`s "Lifeline" subsidiary - which makes and sells specialty cells and growth media - to the point where it will be generating profit (within two years) that they can then plow back into the company's clinical research.

I'm no corporate finance expert but this is what I read from the SEC filing behind the press releases. Andrei Semechkin, Rouslan Semechkin, and X-Master, Inc were the investors putting up the $1M to-date. By way of a "material and essential inducement for the Investors to enter into the Preferred Stock Purchase Agreement, the Company agreed to employ Rouslan Semechkin and Andrei Semechkin in accordance with the terms" of the employment agreements attached to the stock purchase agreement. Andrei is going to be paid at least $180,000 per year for the next 5 years as the company's Chief Business Officer and Rouslan is also on salary.

So, provided ISCO stays around, Andrei and Rouslan will get their $1M back over 5 years in addition to whatever benefits they may glean from their shares. This certainly seems like a nice, low - albeit not entirely without - risk arrangement. On the other hand, this could be seen as them putting their own money in first where they are asking others to follow or they may buck up more money on the subsequent tranches. However this plays out, it is another testament to the fact that in biotech, survivability is so-oft closely if not directly tied to financing creativity!

You likely won't find a press release about this one but J&J is quietly shuttering at least two of their cell therapy programs. Their autologous HIV cell therapy program and their Treg program are both on the out-licensing block after corporate decided to shut down the programs - the former after reportedly 'good' phase II data and the latter even before it went clinical. J&J is not announcing what's behind the decisions though unofficial reports are that it is not a lack of belief in cell therapies overall. Speculation is that it is primarily HQ needing to conserve spending on longer-term programs. Whatever is behind the decisions, it's a little disappointing because for a couple of years now J&J has been quietly been the largest cell therapy company in the world if one added up all their cell therapy internal programs and external investments. They may well still deserve this title but they're a little smaller this month then they were before.

So...since it was a little slow on the industry front, I'll finish with three non-business related items I found interesting this week:

WIRED has published its much-read list of top scientific breakthroughs of the past year and not only do life sciences figure prominently in the top-10 but two of them are cell therapy related. On the list: A new approach to reprogramming stem cells to avoid a tendency to become cancerous; using a bone marrow transplant from an HIV-resistant donor in Germany to eradicate the virus, pointing to gene editing as a possible cure; and using a patient's stem cells to grow a new trachea. The new trachea was number two on the hit parade and earned kudos as the greatest single breakthrough in life sciences.

Adding fuel to the 'cancer stem cell' debate, Canadian researcher Mick Bhatia, and his team, have published a letter abstract in Nature Biotechnology, on a series of tests they claim can distinguish between cancer stem cells and the good kind. Thus enabling, inter alia, the better targeting of cancer stem cells.

Cell Transplantation has become an "open access" journal from the 1st January 2009, starting with volume 18, making it available on the World Wide Web without subscription to researchers and clinicians as well the public and members of the media.

Finally, Dawn Driscoll (DCi Biotech) and I are putting on a 1/2-day course entitled Commercial Considerations for Cell-Based Therapies Feb. 24 at the Moscone Center in San Francisco immediately prior to CHI's Stem Cell Congress (part of their Molecular Medicine Tri-Conference). Let me know if you're interested.

That's cell therapy the way I saw it in this first whole week of 2009...

Friday, January 2, 2009

Cell Therapy Industry HiLites 2009-01-02

Happy new year everyone.

2009 feels like it has the potential to be the somewhat overlooked precursor to the 2010 milestone - not that that's bad just perhaps a "building" year rather than a champagne-popper. Whatever it turns out to be I wish you all a happy and prosperous year.

Based on how slow this week was, I fear next week. I predict a killer backlog of pent-up news on its way. In any event, here's what trickled out this week...

Last week Medistem announced it had filed an IND for use of their new endometrial regenerative cells (ERC) for treatment of critical limb ischemia.

ACT & CHA Biotech have rethought the name for their new joint venture (formerly announced as "Allied Cell Technology" the acronym for which is also 'ACT') - opting instead for the more distinct but lengthy, "Stem Cell & Regenerative Medicine International".

Stem Cell Sciences plc, which is listed in Australia and the United Kingdom announced that it had received a GPB200,000 ($A426,348) loan to use for working capital. In return, the third-party lender has been granted an exclusive period to conduct further due diligence in relation to Stem Cell Science's business and assets. Stem Cell Sciences said it was not in talks with any third parties that might lead to an offer for the issued share capital of the company. Stem Cell Sciences, whose shares have been suspended from quotation since November 26 pending a review of the company's financial position and strategic options, last traded at 15 cents.

Wading now into deep speculation, this tickles the senses like a move Stem Cells Inc might make (I have no information upon which to base this except their past actions) though the SCS assets would not seem to fit with STEM's portfolio. In that respect, this would seem like more of a natural acquisition by Pfizer which did a 5-year deal with SCS in November. I suspect we shall soon see...

Just to make things a little more curious, SCS later announced publication in Cell of a pioneering technique for creating authentic ES cells from rats. They believe the publication to be the first in which germ-line transmission from rat ES cells has been definitively demonstrated. It uses technology licensed exclusively to SCS from the University of Edinburgh and developed by Professor Austin Smith and his team, now at Cambridge University.

Alright this isn't a business item but it has business ramifications and I've discussed this issue in my blog a number of times. "Definite and measurable neurological and ophthalmological improvement." That was the clinical observation of ophthalmologist, Dr. Jack Guggino, in comparing 5-year old Xavier's post stem cell treatment exam results with those of a pre-treatment baseline exam. Xavier, diagnosed at age 2 with optic nerve hypoplasia, was flown to China for a controversial stem cell treatment from the sort of clinic that is drawing much criticism from the global scientific community. These clinics are criticized for, among other things, lack of transparency, formal clinical trials, peer-reviewed publications, and patient follow-up to track long-term clinical results. Recent criticism has suggested results are exaggerated and risks downplayed. Nevertheless, if Xavier were my child what would I do? Ok perhaps it would take something terminal and I'm not saying I would, I'm just sayin'...

Just to polish off the year with a little more successful fundraising news, International Stem Cell Corporation (OTCBB:ISCO) announced it has received the first $1 million tranche of an anticipated private equity financing of up to $5 million to be funded over the next several months. The total amount of the financing is intended to allow the Company to retire its existing secured debt and fund operations of the Company as it moves forward with planned pre-clinical trials in the first quarter of 2009.

Watch for a blog coming soon adding up my totals for how much was raised this year by companies in the space.

In a curious case of the company vs the newspaper, the Seattle Times reported on Dec. 24 that CellCyte Genetics had shut down and was unable to pay rent on its Bothell headquarters citing the company's delayed SEC filing. The company, whose shares trade over-the-counter and in the Frankfurt Stock Exchange, saw its value soar past $400 million last year but since plummeted in the midst of questionable activity currently under investigation and the subject of litigation. CellCyte's shares plummeted in January after The Seattle Times published stories describing the stock-promotion efforts and inconsistencies in the résumé of CellCyte Chief Executive Gary Reys. Clearly the Times and CellCyte are not on talking terms because on Dec. 29 Cell Cyte issued a press release saying the Times had it all wrong citing an Aug. 14 press release in which the Company reported progress in its bioreactor business segment and generally described its development plan to commercialize its bioreactor product line. CellCyte reiterated its intent to execute this development plan in 2009.

The company's Form 10-Q, filed Dec. 22, states:
"As of September 30, 2008, the Company had a working capital deficit of $897,402, and an accumulated deficit of $9,890,982 incurred through September 30, 2008. The Company has no revenues. Management forecasts operations during the coming year will need approximately $2,500,000 in additional working capital, which is expected to come from issuance of convertible notes and private placements of stock."

The filing laid out detailed plans for the company over the next 12 months but also stated the following: "As of September 30, 2008, we had cash in the amount of $5,734 and a working capital deficit of $897,402. Our planned expenditures over the next 12 months are expected to amount to approximately $2,500,000 and will exceed our cash reserves and working capital. We presently do not have sufficient cash to fund our operations and have curtailed significantly all activities. We anticipate that we will require additional financing in order to pursue our plan of operations for the next 12 months. There can be no assurance that we will obtain any additional financing in the amounts required or on terms favorable to us. If we are unable to obtain additional financing, we may have to abandon our business activities and plan of operations."

The Seattle Times may be guilty of a little prognostic reporting here but it certainly does seem like CellCyte will have to pull a wee little rabbit out of the hat to keep things going. Having said that, I've certainly heard the premature predictions of the imminent demise of many a cell therapy company before only to watch them 'pull a phoenix'. I like it when that happens.

I don't pretend to fully understand the Cytori patent portfolio and after the legal wrangling settled who ended up with what for what but Cytori announed the issuance of U.S. patent # 7,470,537 (the ‘537 patent) covering adipose-derived stem and progenitor cells to the University of California. The patent application containing these claims is jointly owned by the University of California and the University of Pittsburgh. The composition of matter patent is licensed to Cytori through its agreement with the University of California. The cells covered by the issued claims are believed to encompass a clinically important subpopulation of cells within adipose tissue.

BIO says one in four of the 370 publicly traded U.S. biotech companies have less than six months of cash on hand. But it's not just industry that's impacted by the economic crunch. The global financial crisis may do what opponents of California’s $3 billion state-sponsored stem cell research experiment could not: dry up funding. To keep the money flowing, later this month CIRM will weigh a contingency financing plan that could include bond anticipation notes and a private placement with major philanthropic backers.

And that brings me to my challenge to you all. I know money is tight but it is even more important in times like this that we remember to support the non-profits that support life as we know it for so many. This is true in your local community, on a global scale and it is also true in the industry in which you work. The non-profit organizations that provide critical support to the cell therapy industry need your financial support and your volunteer contributions. This week I announced that I've committed the Cell Therapy Group to supporting ISCT by way of a 2009 corporate membership. Please consider doing the same.

And that, my dear readers, polishes off 2008 and the first couple days of 2009. That's the cell therapy industry the way I saw it over the holiday season.

As Robert A. Preti is fond of saying... "Be careful out there".