Cell Therapy Industry HiLites 2009-03-25

I am more intrigued every day by the potential for identifying opportunities in the convergence of cell therapies, diagnostics, personalized medicines, theragnostics, regenerative medicine, and more traditional biotechnologies and pharmaceuticals.

I wonder to what extent we will look back on these primitive days of ex vivo cell therapy and chuckle at its ephemeral but educational existence much like betavision or pervasive therapeutic bloodletting. I suspect many of these therapies will have a role in future medicine but I'm starting to believe more and more that the future of cell therapy will be more about in vivo manipulations than ex vivo manufacturing.

In an attempt to catch up from my brief hiatus and to have more frequent and less tome-like posts, I bring you this mid-week edition of Cell Therapy Industry HiLites.

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Contact Lee [at] celltherapygroup [dot] com

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FINANCIAL

Given the continued extraordinary market conditions, NASDAQ has further suspended enforcement of the rules requiring a minimum $1.00 per share. closing bid price and a minimum market value of publicly held shares until Monday, July 20, 2009. Good new for many including Aastrom Biosciences, Inc. (Nasdaq:ASTM) which now has until September 18, 2009 to regain compliance with the $1.00 minimum closing bid price rule in order to remain listed on the Nasdaq Capital Market.

As promised, International Stem Cell Corporation (OTCBB:ISCO) has received the third $1 million tranche of an anticipated private equity financing of up to $5 million the remainder of which is says will be funded over the next several months.

Now that Cell Genesys, Inc. (NASDAQ:CEGE) has no products to develop things are looking up for it! The company reported financial results for the fourth quarter and full year ended December 31, 2008. The company reported a net loss of $13.3 million ($0.15 per fully diluted share), for the fourth quarter of 2008, compared to a net loss of $33.4 million ($0.43 per fully diluted share) for the corresponding period in 2007. The net loss for the year ended December 31, 2008 was $47.0 million ($0.56 per fully diluted share), compared to a net loss of $99.3 million ($1.39 per fully diluted share) for the year ended December 31, 2007. As of December 31, 2008, the Company had $86.1 million in cash, cash equivalents and short-term investments. The Company currently expects to have approximately $73 million and $69 million in cash, cash equivalents and short-term investments at March 31, 2009 and June 30, 2009, respectively. The Company continues to explore strategic alternatives, including merger with or acquisition by another company, further restructuring, allocation of its resources toward other biopharmaceutical product areas, and sale of the Company’s assets and liquidation of the Company.

CLINICAL

As a result of ongoing and active discussions in our LinkedIn Cell Therapy Industry Group, it has come to my attention thatprivately-held Harvest Technologies Corp - a company known for its bone marrow and platelet concentrate devices - has entered into the therapeutics business. It recently obtained an Investigational Device Exemption (IDE) from the FDA to conduct a randomized, controlled, double-blind, multicenter clinical trial using the Company’s BMAC System to prepare a composition of bone marrow stemcells to treat patients with Critical Limb Ischemia (CLI). This “feasibility trial” will enroll a total of 48 subjects. Subjects who enroll in this study will have to have exhausted all surgical and procedural options and are at extreme risk for major amputation. The BMAC System is a point-of-care device for concentrating a patient’s own (autologous) bone marrow stem cells in approximately 15 minutes at the bedside. The clinical study design provides for injecting these cells into the affected limb to reduce the potential for limb amputation. It also will offer data with respect to the safety of this procedure.

What's even more interesting is that reportedly the company has submitted an investigation device exemption for a double-blind placebo cardiac safety study—to inject BMAC cells into the myocardium during bypass surgery in patients with severely compromised heart function.

This presents an interesting regulatory pathway for a couple of business models both in providing point-of-care devices and providing therapeutic service employing such devices. More on that in an upcoming blog (and in our LinkedIn discussions).

The Indian Council of Medical Research and the Drug Controller General of India is said to have now approved protocols Bangalore-based firm Stempeutics Research for multi-center phase I/II double blind randomized clinical trials of bone marrow derived ex vivo cultured adult mesenchymal stem cells for Elevated Acute Myocardial Infarction and Critical Limb Ischemia. The cells are to be administered by injection.

COMMERCIAL

MultiCell Technologies, Inc. (OTC Bulletin Board: MCET - News) announced it has entered into a cooperative research and development agreement with Maxim Biotech, Inc. to develop products for the study of liver stem cells and liver cancer. The cooperative research and development agreement with Maxim Biotech, Inc. will initially focus on the development of a family of life science research reagent tool kits which can be used to isolate liver stem cells, and help to elucidate liver stem cell gene function and their encoded proteins. MultiCell plans to further leverage this research effort involving liver stem cells to identify therapeutic targets, and diagnostic and prognostic markers of liver cancer. MultiCell will also seek to develop and patent therapeutic product opportunities specifically targeting the treatment of primary liver cancer and intrahepatic bile duct cancer.

Kosdaq-listed RNL Bio (famous for dog cloning) faces a KFDA ban on its cosmetic containing human-derived stem cells.

J&J's Therakos, Inc. announced the U.S. Food and Drug Administration (FDA) approval of the THERAKOS™ CELLEX™ Photopheresis System for the palliative (reducing the severity of symptoms) treatment of the skin manifestations (appearance) of cutaneous T-cell lymphoma (CTCL) that are unresponsive to other forms of treatment. The THERAKOS™ CELLEX™ Photopheresis System is a system that uses extracorporeal (outside the body) photopheresis (ECP) cell therapy to relieve the symptoms of CTCL. The system also has been cleared recently in Canada and Europe. The new THERAKOS™ CELLEX™ Photopheresis System is said to feature several improvements over but retain the primary benefits of the predecessor THERAKOS™ UVAR™ XTS™ Photopheresis System. The improvementa are said to open up this treatment option to patients for whom it was previously unfeasible including lower weight patients.

Stem Cell Therapy International, Inc. (OTCBB: SCII) decided it was worthwhile to announce that they are in "strategic meetings" with unnamed "global stem cell representatives" and they are "not wasting any time...with the merger of Stem Cell Therapy International, Inc. and Histostem, Ltd. of South Korea" to create the newly name company, AmStem International. Now that's great news.

StemCelDigest.net outlined the single-digit degrees of similarity between companies (STEM, ISCO, ACT) pursuing stem cell solutions for diseases of the retina. Other companies in this space include the Pfizer-backed EyeCyte and UK's ReNeuron.

RESOURCES & EVENTS

Watch for an announcement from ISCT announcing a call for late-breaking abstracts.

Jon Rowley (Lonza) reports that there is more stem cell content - from science to manufacturing - at BIO2009 this year. Dare we think it's not a bubble this time?

Sign-off

A proposal to change the name of the American Society of Gene Therapy (ASGT) to the American Society of Gene and Cell Therapy (ASGCT) has been nanimously approved by the Board of Directors and the proposal is now being forwarded to the general membership of the Society for a final vote in April of 2009. In an editorial in December issue of Molecular Therapy, Membership Committee Chair and Past ASGT President, Savio LC Woo, stated that the Membership Committee has recommended that the following concrete steps,among others, be implemented to maximize its positive impact.

• First, the term “cell therapy” must be included in the Society’s mission statement.
• Second, the Society’s leadership should contact the leadership of the International Society for Cellular Therapy (ISCT) to assure them that our name change represents an opportunity for both societies to collaborate and advance our common interests and to initiate a dialogue with them onthe needs and interests of cell therapists so as to better recruit these researchers into our society.
• Third, the Society leadership should reach out to leading investigators and the research communities in adult and ES cell biology as well as iPS cell biology.
• Fourth, the Program Committee should ensure that cell therapy is richly represented in the symposium programs of the Society’s annual meetings.
• Fifth, the Nominating Committee will need to make a concerted effort to identify and nominate prominent cell therapists into the Society’s leadership positions.
• Finally, the editorial team of Molecular Therapy—the Society’s official journal—is encouraged to increase even further the important efforts they have made over the past few years to recruit Editorial Board members with expertise in cell therapy and stem cell engineering, in additionto continuing their successful solicitation of outstanding articles in these areas.
  

Professional organizations, it seems to me, are facing the collective pressures of increasing financial constraints, the rise of online access to publications minimizing the need for member-rate subscriptions, and the networking power of online social networks, all combining to make their value proposition much less obvious to many - particularly the younger set.

I understand ASGT's desire to ensure its mandate stays relevant and inclusive of all aspects of therapies employing genes. I also believe the increasing overlap between organizations representing "cell therapy" is not in the best interests of the sector. While ISCT remains the only organization with cell therapy of all types at the core of its mission, AABB has encroached into the cell therapy space from the blood banking side, ISSCR from the basic stem cell research end of the spectrum, ASBMT from the clinical stem cell transplant side, TERMIS from the tissue enginering, and now ASGT (following the precedent set by ESGT-ESGCT) from the gene therapy end.

As C. Mason & P. Dunhill wrote last year in Regenerative Medicine, what the industry needs is a strong voice generated by a powerful industry association. As long as we continue to create more silos of representation, we will continue to stray farther away from what's needed. While I have strong ties to ISCT and continue to serve that organization in several capacities, I am increasingly frustrated by the dilution of this nascent industry's time, money, and efforts spread over too many organizations and conferences without creating the voice for industry that is needed.

I'm not sure where this frustration will take me but my increasing conviction is that the companies in the cell therapy sector are ill-represented in any organization and spattering attempts at representation throughout all of them (including BIO which has to-date all but ignored the sector) is increasingly insufficient.

Am I off base here? Is this a real need or am I making something out of nothing? What do you think?