Cell Therapy HiLites 2008-12-05

While the cell therapy sector certainly has more than its fair share of companies spreading their peanut butter thinly to survive their burn rate until their next financing closes, another week of funding news and milestones means its not all bad news out there for the cell therapy industry.

Pluristem Therapeutics Inc. (NasdaqCM:PSTI) announced $750,000 of new investment funds - well actually $600,000 from a restricted share purchase and an additional $150,00 from management and BOD members by way of reduced compensation. But the investor has the option, within 10 business days following the initiation of the Company's first human clinical trial, to put in another $600,000.

Osiris completed enrollment in its first Phase III stem cell trial - this one studying Prochymal for steroid refractory acute GvHD - encompassing 244 patients at 74 centers in 8 different countries (N. America & Europe). The last patient is expected to complete the trial in May 2009. MSCs have been reported as an effective agent in the treatment of GvHD by a number of medical centers. In a recent Lancet publication, Le Blanc et al. reported a 55% complete response rate when using MSCs in the treatment of steroid-resistant GvHD. Many people are watching with keen interest to see how the big questions around Osiris' BLA/MAA submissions/approval and then (hopefully) commercial issues like IP, reimbursement and product revenue models will be addressed as Osiris gets ever-closer to market.

Osiris also received approval from Health Canada to expand access to Prochymal for pediatric patients in Canada with GvHD following a similar ruling earlier this year by the FDA. Under the expanded access program, children 2 months to 17 years in age inclusive with Grades B-D acute GvHD not responsive to steroids are eligible for treatment.

Biocompatibles' CellMed treated their first stroke patient in a phase I/II trial with gene-modified, adult, allogeneic, alginate-encapsulated, MSCs dipped into the brain for 14 days (and then removed). The trial is described as the first "authorized" trial using stem cells for stroke.

In this issue of Cell Stem Cell, an ISSCR Commentary article outlines their proposed Guidelines for the Clinical Translation of Stem Cells, available in full online at the ISSCR website. The recommendations include an insistence on expert evaluation and independent oversight, a thorough informed consent process, and transparency in reporting of clinical trial results. This is aimed at all those stem cell transplant clinics selling direct to consumers with little - if any - regulatory oversight, formal clinical trials, or peer-reviewed publications.

Complementing the release of the Guidelines, in a Correspondence article, stem cell law and policy professor Tim Caulfield and colleagues published an assessment of the online portrayal of commercial stem cell transplantation retail ventures providing offshore treatments. Conclusion? They reveal most such clinics exaggerate claims and omit risks.

Organogenesis announced the receipt of two awards honoring the company's development, manufacturing and economic impact. It's been a long and twisty road for the company reinventing itself from Chapter 11 (filed in 2002) but it's certainly now the real deal and a somewhat unheralded beacon for regenmed.

On the not-so-bright side, Cell Genesys, Inc. (Nasdaq:CEGE) and Takeda Pharmaceutical Company Limited (TSE:4502) announced the termination of their collaboration agreement for the development and commercialization of GVAX Immunotherapy for prostate cancer. Takeda will return all commercial rights to GVAX immunotherapy for prostate cancer to Cell Genesys and make certain wind-down payments to Cell Genesys in connection with the phasing out of the remaining clinical development activities.

In keeping with their unique approach to life ACT has formed another ACT. Worcester biotech Advanced Cell Technology Inc. and Korean biotech CHA Biotech Co. Ltd. have formed a new stem cell technology development company called Allied Cell Technology, to be based - wherelse? - in Worcester. The international joint venture will use ACT’s hemangioblast cell technology to develop human blood cells. Allied Cell Technology will be majority owned by CHA, with ACT (OTC: ACTC) licensing its technology for a $500,000 license fee from CHA. Leading the new company in Worcester will be Young Chung, as well as Shi-Jiang Lu and the ACT hemangioblast team. Robert Lanza of ACT will serve as the chief scientific advisor of Allied Cell Technology.

Katarina LeBlanc of Karolinska Institute has been awarded a $1.2 million prize for her work in "immunological characteristics of mesenchymal cells and their use in mesencymal stem cell therapy". The prize, funded by the Tobias Foundation, will be awarded December 17.

Another wannabe cell therapy CMO takes the exit stage right - I think? WuXi-Apptec intends to close its US biologics manufacture operations to focus on testing. Chalk this up to another of several over the past handful of years who have entered - and shortly thereafter exited - the cell therapy CMO business. It's not an easy game and the true players are in it for the long-term. While they announced Wuxi-Apptec will no longer be doing "biologics manufacturing" in the US they said they are now going to focus on "cell therapy". There seems to be some confusion about what exactly that means. I guess we'll stay tuned.

Finally found a half-decent and relatively objective analysis of the recent European Patent Organization (EPO) ruling on the WARF patents sans spin.

On the gene therapy front, Ceregene reported clinical data from a double-blind, controlled Phase 2 trial of CERE-120 in 58 patients with advanced Parkinson’s disease. Regrettably the trial did not demonstrate an appreciable difference between patients treated with CERE-120 versus those in the control group. Continuing the theme, Introgen Therapeutics, Inc. (NASDAQ:INGN) announced that as part of its ongoing restructuring, the company has elected to file Chapter 11 petitions in the U.S. Bankruptcy Court. The EMEA has accepted, from Introgen's wholly-owned subsidiary in Ireland, Gendux Molecular Limited, a Marketing Authorization Application (MAA) for Introgen's ADVEXIN to treat recurrent, refractory head and neck cancer. However in September 2008, the FDA notified Introgen that the BLA was not sufficiently complete and would not be filed at that time. Demonstrating once again that success and failure are a hair's breadth apart.

HemoGenix announced the implementation of biorisk-free, green-source growth factors and cytokines for use in all HALO® Assay Kits and HALO® Contract Research Services

CBER has been busy these past few months. FDA released another new Guidance this week. This one: Guidance for Industry: Cooperative Manufacturing Arrangements for Licensed Biologics.

NovaRx Corporation appointed former CSO of Invitrogen - remember them? - Norrie J.W. Russell, Ph.D as President and COO this week. His pedigree should be invaluable as NovaRx manages the late-stage development of it lead product candidate, Lucanix(R) - a whole cell-based therapeutic vaccine comprised of four allogeneic cell lines, currenly in Phase III clinical trial for the treatment of progressive non-small cell lung cancer. Once fully open, the trial is expected to run in 90 centers in the US, Canada, Serbia, Hungary, UK, Netherlands, Germany, Poland, Italy, and India.

And finally, on the mere speculation front, yet another analyst has identified Genzyme as a ripe acquisition target by one of those cash-rich, pipeline-poor pharma's on a shopping spree.

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