Cell Therapy Industry HiLites 2009-02-27

I'm pretty excited by how well the blog is being received. This is a small, niche segment of the biotech sector and I only cover the commercial aspects of the segment so the numbers are small but - as you can see from the graph on the right - the number of people checking out the blog is certainly growing exponentially. This month we passed 1200 visit and 2200 views. Thanks for spreading the word...

Apparently this is the week for which we've been waiting for 40 years. Peter Winter reports to the Burrill Report that the biotechnology industry is now profitable.

The 360 publicly listed companies they track generated $9.4B in revenue in 2008 with a net income of $3B. 67 of these companies companies were profitable with the remainder generating red ink of $6B. The bulk of the income was, of course, generated by the top 3: Genentech, Amgen, Gilead - reporting a combined net income of $8B. 200 of the companies are in fiscal trouble with many having only 6-12 months of cash left. Peter speculates one-third of the companies being tracked today may not exist as-is one year from now - perhaps to the benefit of a stronger sector.

FINANCIAL

The VC panel at the Stem Cell Congress (CHI's Molecular Medicine Tri-Conference) in San Francisco this week was asked whether - if they were given it - they would take $500M to invest over a 5-year term and get out over the next 5 years with the 'usual' VC expectations of return. Only one string: the investments would have to be restricted to regenerative medicine companies (defined widely to include therapeutics, tools, devices, platforms, reagents, etc). Tellingly, they all answered 'no' or that they would take only part of the money. They were all VCs very familiar with the space and even personal cheerleaders for it but they all felt it was too early to find enough companies they could invest that much money in with confidence of giving the requisite returns. I'll be exploring this more in blogs to come.

With that, the only official financial news is not an investment but a loan. Wisconsin Governor Jim Doyle announced that Stratatech Corporation in Madison will receive a $500,000 loan from the Department of Commerce.

Unofficially, executives from SanBio, Inc confirmed for me this week that they have received a small (hopefully precursor) investment from Fujifilm Life Science. Amounts were not disclosed. This reportedly modest financing is intended to get SanBio through its IND approval. The company is actively fundraising.

CLINICAL

Angioblast Systems Inc announced the successful achievement of the key safety milestone in the first low-dose cohort of patients treated with Revascor(tm), the proprietary allogeneic, or "off-the-shelf", universal adult stem cell product for congestive heart failure. Safety data from all 20 congestive heart failure patients enrolled in the multi-centre Phase 2 clinical trial were reviewed by the trial's Data Safety and Monitoring Board (DMSB). No cell-related adverse events occurred in any patient during the 30-day follow-up period and the review was positive. As a result, the DSMB has allowed the company to move forward with recruiting the second 20-patient cohort of patients with congestive heart failure. This group will receive a higher dose of cells.

The placebo-controlled trial of Angioblast's Revascor(tm) cell therapy will randomise up to 60 patients suffering from congestive heart failure to three 20-patient cohorts receiving either a progressively increasing dose of the company's allogeneic adult stem cells or standard of care. The cells are implanted into the damaged heart muscle using the NOGA MyoStar catheter technology system provided through a collaborative agreement with Johnson & Johnson companies, Biologics Delivery Systems and Cordis Cardiology. The outcomes in each patient group following injection of progressively increasing doses of cells will be compared against standard-of-care in terms of both safety and effectiveness at halting or reversing congestive heart failure. The company will provide interim efficacy results for each dose cohort as they become available.

Cytori Therapeutics, Inc. (NASDAQ: CYTX) released a press release entitled "Adipose-Derived Stem and Regenerative Cells Prevent Mortality and Reduce Kidney Damage in Preclinical Study"preclinical results". The opening line of the reiterated that the data reported were "preclinical results". The release also clearly stated the "study was performed using a manual extraction technique" though it did speculate that the "potential application could be made more efficient and cost effective with the use of Cytori's Celution(R) System to isolate clinical grade ADRCs in real time".

StemCellDigest.net, however, was critical of the release in a blog entitled "Now You See It, Now You Don't" - The Latest Cytori Press Release" calling the company one of the most "flamboyant" in the sector and its communications style and treatment of the facts as taking "liberties with unwritten research release protocol that is a bit beyond the pale". They said: "Quickly read, the release seems to suggest that this was a clinical study". Now I understand the comment that Cytori certainly seems aggressive in its communications style but one would have to read this latest press release awfully 'quickly' to believe it was data from a clinical study despite the references to animals (mice?) as 'subjects' - admittedly not a standard practice.

Intercytex Group Plc (AIM: ICX) has products on the market and other products in its phase II pipeline but it has recently trimmed a significant amount of staff to cut back on costs and now in a recent stock exchange statement the company states it is going to abandon its only phase III trial - that of Cyzact® for venous leg ulcers. Indeed "no further work on Cyzact® is planned in any indication" and "in the light of this disappointing result the board has determined to review all strategic options for the company" including "a possible merger or sale of the business".

For the second time this month, we've had a press release with the general theme of: 'we're closing up shop now...hope we contributed to the science'. At the ASCO meeting this week Cell Genesys, Inc. (NASDAQ:CEGE) announced results from further analyses of their prematurely closed VITAL-1 and VITAL-2 clinical trials studying GVAX immunotherapy for prostate cancer. Both trials were shut down earlier this year and the company is being cratered.

VITAL-1 was a Phase 3 clinical trial designed to compare GVAX cancer immunotherapy as a monotherapy to Taxotere chemotherapy plus prednisone in castrate-resistant prostate cancer (CRPC) patients with metastatic disease who were asymptomatic with respect to cancer-related pain. The primary endpoint of the trial was an improvement in survival. In 2007, the VITAL-1 trial completed enrollment with 626 patients at 131 sites in North America and the European Union. In January 2008, Cell Genesys announced that the Independent Data Monitoring Committee (IDMC) had completed a pre-planned interim efficacy analysis for VITAL-1 and recommended that the study continue, providing no further information to the Company other than the recommendation to continue the trial. On August 27, 2008, the Company announced that it had requested the IDMC to conduct a previously unplanned futility analysis of VITAL-1. VITAL-1 was terminated in October 2008 based on the results of a futility analysis conducted at the Company’s request by the study's IDMC which indicated that the trial had less than a 30 percent chance of meeting its predefined primary endpoint of an improvement in overall survival. Based on the results of that analysis, the Company terminated the VITAL-1 trial in October 2008.

The final Kaplan-Meier survival curves for the two treatment arms suggest a late favorable effect of GVAX immunotherapy on patient survival compared to chemotherapy, with the curve for GVAX patients crossing above the chemotherapy curve at approximately the same time median survival was reached in both treatment arms (21 months). Additionally, the data suggest that patients with Halabi predicted survival (HPS) greater than or equal to 18 months may have a more favorable response to the immunotherapy. Treatment with GVAX immunotherapy was generally well-tolerated and had a very favorable side-effect profile compared to Taxotere chemotherapy particularly with respect to a lower frequency of grade 3 or higher toxicity of nine percent versus 43 percent.

VITAL-2 was a Phase 3 trial designed to compare GVAX immunotherapy in combination with Taxotere to Taxotere plus prednisone in CRPC patients with metastatic disease who were symptomatic with respect to cancer-related pain. The primary endpoint of the trial was also improvement in survival. VITAL-2 was initiated in June 2005 and had enrolled 408 patients at 115 clinical trial sites located in North America and the European Union prior to study termination. On August 27, 2008, the Company announced its decision to terminate enrollment and treatment with GVAX immunotherapy in VITAL-2 as recommended by its IDMC which, in a routine safety review meeting held at that time to review both VITAL-1 and VITAL-2, observed an imbalance in deaths between the two treatment arms of the VITAL-2 study.

Updated analyses show no significant toxicities in the GVAX plus Taxotere arm that could explain the imbalance in deaths. Eighty-five percent of deaths were reported as due to prostate cancer in both arms, and there was no trend in the causes of death in the remaining patients. These observations are consistent with the hypothesis that the decision to omit concomitant prednisone in the GVAX immunotherapy treatment arm to avoid the immunosuppressive effects of prednisone may have contributed to an unfavorable outcome compared to the combination of chemotherapy and prednisone. Additionally, it is important to note that further analyses of VITAL-2 have indicated that the imbalance in deaths between the two treatment arms has decreased from 20 deaths as reported at that the time of the IDMC’s initial analysis (August, 2008) to 9 deaths at the time of the final analysis (December, 2008).

COMMERCIAL

Osiris Therapeutics, Inc. (NASDAQ:OSIR) announced its results for the fourth quarter and year ended December 31, 2008. It's list of recent and 2008 highlights is admittedly impressive:
* Formed major strategic alliance with Genzyme Corporation worth up to $1.4 billion for the development and commercialization of Prochymal and Chondrogen in countries outside the United States and Canada.
* Sold the Osteocel business to NuVasive, Inc. in a transaction worth up to $85 million in upfront and milestone payments.
* Awarded Department of Defense contract fully valued at $224.7 million to develop and stockpile Prochymal for acute radiation syndrome (ARS).
* Received approval to initiate Prochymal expanded access program in the US for adult and pediatric patients and in Canada for pediatric patients suffering from life-threatening Graft versus Host Disease (GvHD).
* Completed enrollment in first worldwide Phase III stem cell clinical trial for the treatment of steroid-refractory GvHD.
* Reported positive two-year data from Phase I clinical trial evaluating Prochymal in heart attack patients.
* Completed enrollment of Phase II clinical trial evaluating Prochymal in patients with chronic obstructive pulmonary disease (COPD).
* Reached agreement with the FDA regarding the timing and content of the submission of the first marketing application for a stem cell product.
* Reported cash, short-term investments and receivables of $123.5 million at year-end.

Genzyme Corp.'s chief executive Henri Termeer said the company may spend about $600 million this year to acquire products that treat chronic diseases - likely personalized drugs, highly specialized medicines already tested in humans. He prefers, he says, the idea of buying products rather than entire companies.

Despite being on the verge of closing a multi-million dollar financing, expecting to launch a commercial product next month, and reporting promising findings on the her company's experimental hair regrowth treatment last week at the 4th Annual Stem Cell Summit in New York, Histogen, Inc founder and CEO Gail Naughton has just had to fire all her employees. A patent infringement lawsuit filed last month against Histogen has triggered a funding crisis at the San Diego biomedical startup, which was forced to lay off all 36 of its employees at the end of January. The suit filed by rival SkinMedica of Carlsbad, CA, also prompted a group of angel investors to withdraw their planned $2.4 million investment in Histogen at the end of January. Naughton said the lawsuit was filed to stop the planned March 6 launch of the cosmetic products.

ThermoGenesis, Corp. (NASDAQ:KOOL) announced that the FDA has agreed with its plan to remedy the voluntary recall of certain lots of AXP disposable bagsets that it announced in November. At that time, the Company had indicated that the recall was not the result of any safety issues; the field action was a result of the potential for particulates to be released into the sterile, non-pyrogenic fluid path as a result of a defective component provided by one of the suppliers to the Company. The Company indicated that it was providing customers a blood filter to be used, as necessary, with any bags that had already been processed.

Cordlife Ltd (ASX: CBB) reported its 2008 year end financial results. Cconsolidated revenue and other income for the half-year ended 31 December 2008 increased by 78% to $13,218,000 from $7,439,000 for the half-year ended 31 December 2008. Revenue from cord blood banking services was $11,436,000 for the half-year ended 31 December 2008 as compared to $7,006,000 for the half-year ended 31 December 2007 (an increase of 63%). This substantial growth is due to significant increase in client sign-ups across the Group's existing markets of Singapore and Hong Kong. Several marketing and promotional measures were put in place in the first half of this financial year in our two largest markets. The additional sign ups in Singapore are also due to incentivised schemes from the Singapore government to aid first time parents financially. There was also growth across the Group’s other markets in Australia, as well as Indonesia. India registered its first clients and revenue in the first half of this financial year. Net profit attributable to members for the half-year ended 31 December 2008 was $1,590,000, an increase of 997% over the net profit attributable to members of $145,000 for the half-year ended 31 December 2007.

Cord Blood America, Inc. announced an agreement with Shelter Island Opportunity Fund to restructure its current outstanding debt. With the signing of the agreement, Cord Blood America projects it will become cash flow positive for the first time in its history. The loan originally helped CBAI to acquire a large customer base with the CorCell acquisition. The principals of Shelter Island Opportunity Fund worked with us to restructure our debt, putting us in position to run as a cash flow positive organization. "This is a gesture of true partnering with CBAI, while sharing the stem cell vision to become a globally dominant stem cell storage company", said Matthew Schissler, CBAI Founder and CEO.

BioTime, Inc., (OTCBB:BTIM) announced that its wholly-owned subsidiary Embryome Sciences, Inc. has entered into an agreement with Reproductive Genetics Institute (RGI) of Chicago, Illinois granting Embryome Sciences rights to market new human embryonic stem cell (hES) lines selected by Embryome Sciences from 294 hES lines derived by RGI. Embryome Sciences will initially select 10 RGI hES cell lines, and may add additional cell lines at its option. RGI is a leading fertility center that screens embryos for genetic disorders, such as cystic fibrosis and muscular dystrophy prior to implantation. The RGI hES lines include both normal cells and 88 cell lines identified as carrying a host of inherited genetic disease genes that Embryome Sciences plans to sell as research products to universities and pharmaceutical companies.

NeoStem, Inc. (NYSE Alternext US: NBS) announced it has signed a license agreement to obtain the exclusive worldwide rights to innovative stem cell technology and applications for cosmetic facial and body procedures and skin rejuvenation. This "innovative stem cell skin rejuvenation procedure" is intended to enhance NeoStem's leadership in the anti-aging and regenerative medicine arena - one of their "core pursuits".

Voted one of 2008's Top Ten small companies (<1,000 href="http://www.beaker.com/CompanyProfile.aspx?uid=33e3fa99-db71-45f6-b9c8-165e63e9a633">Tengion, Inc was a Feature Company this month on the Beaker.com. T engion is a clinical stage regenerative medicine company focused on developing neo-organs and neo-tissues derived from a patient's own (autologous) cells. The Company's lead product, the Tengion Neo-Bladder™, is currently in Phase II clinical trials in the United States in pediatric patients with spina bifida, and adult patients with spinal cord injuries.

Cell isolation/collection companies join forces - Gahaga Biosciences has been acquired by Hemacell Perfusion.

RESOURCES & EVENTS

All the articles are available for free download in a special issue of MAL's Tissue Engineering Part A: Technologies for Enhancing Tissue Engineering: Materials and Environments for Guiding Stem Cell Function. Guest Editors are E. Alsberg and J.A. Rowley.

Two prominent supporters of stem-cell research said they had reintroduced a Senate bill that would allow federal financing for human embryonic stem-cell research, in anticipation of President Obama’s support for the work. The advocates, Senators Tom Harkin, left, Democrat of Iowa, and Arlen Specter, Republican of Pennsylvania, said their measure would allow federal financing for research using stem cells taken from human embryos left over from fertility treatments. “It is the same bill that both houses of Congress approved in 2007, but was vetoed by President Bush,” they said. Mr. Obama has promised to overturn Mr. Bush’s policy strictly limiting the use of federal money for such research.

Proposed legislation which would allow companies to grab a share of the $18 million stem cell fund, is pitting the Maryland's biotechnology companies against academic researchers.

Sign off...

In the long-term, one of the take-homes from this week's conference in San Francisco is continued encouragement by the outside players now actively strategizing about how they will participate in the cell therapy industry. This will (a) inject cross-disciplinary intelligence, experience, and maturity into the industry, (b) allow companies in the sector to leverage expertise from outside the sector that can be used to take companies and technologies to the next level of commercial viability, and (c) represent an increasing number of potential M&A partners.

In the short-term, there was nothing to refute the general sense that this is going to be a long and game-changing year for many companies in the sector.

In the meantime, CellTherapyBlog will bring you the good, the bad, and the ugly from the cell therapy and regenerative medicine industry...