What follows is an interesting but not exhaustive list of cell therapy industry clinical milestones we anticipate in the next 3-9 months as selected from the list of cell therapy products we are tracking in late-stage or post-commercial development.
There are other commercial milestones we are monitoring as well as other clinical milestones we expect to see related to cell therapy products in earlier stages of the development pipeline that are not included below.
CellCoTec (www.cellcotec.com)
- Having completed a trial in Europe of their device to enable POC production of an autologous chondrocyte cellular product in/with a biodegradable, load-bearing scaffold for the treatment of articular cartilage defects, they have now submitted their CE market application. The CE mark application is under review and they anticipate a response in October.
- This device and the potential emergence of Sanofi's MACI in the European market next year may have an impact on Tigenix (EBR:TIG) most directly.
ERYtech Parma (www.erytech.com)
- Their 'pivotal' phase 2/3 trial in Europe of lead product, GRASPA, for the treatment of Acute Lymphoblastic Leukemia (ALL) is scheduled for completion 2H 2013.
GamidaCell (www.gamidacell.com)
- Their 'pivotal' phase 2/3 trial in the US, Israel, and Europe of lead product, StemEx, for the treatment of leukemia and lymphoma, in joint development with Teva, completed enrollment in February and is scheduled for completion 2H 2012. They have not been shy about the fact they expect to be in the market in 2013.
Innovacell (www.innovacell.com)
- They raised over 8m Euro in April for a phase 3 trial in Europe for their lead product, ICES13, for the treatment of stress-urinary incontinence which was scheduled for a preliminary clinical data readout in Q4 2012 and be ready for market authorization in 2013. Since announcing the capital raise the company has been stone silent and no clinical trial registry has been filed. Status unknown.
Miltenyi Biotec (www.miltenyibiotec.com)
- Their phase 3 trial in Germany of CD133+ cells as an adjunct to CABG surgery for myocardial ischemia or coronary artery disease is scheduled for completion in January.
NovaRx (www.novarx.com)
- Their phase 3 trial in US, Europe, and India of their lead product, Lucanix, for the treatment of advanced Non-small Cell Lung Cancer (NSCLC) following front-line chemotherapy is scheduled in clnicaltrials.gov for completion in October but we have learned they expect their next 'interim analysis' in February.
NuVasive (www.nuvasive.com)
- They have a series of trials scheduled to complete 2H 2012 intended to provide additional clinical data to support its marketing of Osteocel Plus for the treatment of a growing number of orthopedic applications.
Sanofi's Genzyme (www.genzyme.com)
- Having completed their phase 3 trial in Europe of MACI for knee repair (symptomatic articular cartilage defects of the femoral condyle including the trochlea), they expect to file their market authorization application (MAA) in 1H 2013.
Hope that's helpful and gives you a sense some of the late-stage things to watch for in the coming weeks and months.
Rumor is there was little in the way of cell therapy at the ASH meeting this year and the TERMIS meeting was mostly "basic research". I'd love to hear other reports. Maybe because of that, it seemed oddly slow on the cell therapy front this week. Despite that, we've got some interesting news to review.
This issue of Newseek had an article by David T. Scadden, M.D. and Anthony L. Komaroff, M.D. "Will Stem Cells Finally Deliver? Major breakthroughs are propelling the field. Science becomes medicine."
TiGenix secured grant support to deepen its pipeline. The company has been awarded a €1.81 million, or $2.41 million, grant to support its R&D efforts a meniscus repair program. The two-year program will focus on the further development and application of allogenic stem cells in combination with a biocompatible scaffold, the identification of biomarkers predictive for the quality of meniscus-like tissue, and establishing preclinical proof-of-principle for cellular repair of meniscus lesions.
The European registration procedure for its lead product, ChondroCelect®, is reportedly progressing. As this is the first cell based product submitted to the EMEA under the central registration procedure, some further documentation on the product and process validation processes was recently requested by the regulatory authorities. The company is apparently now compiling further information for submission, and expects the CHMP to issue an opinion on the marketing authorization for ChondroCelect in the first half of 2009. If approved, ChondroCelect will be the first cell-based product to receive a central marketing authorization for an advanced therapy medicinal product in Europe. The preparation of the Biologics License Application (BLA) to obtain product registration in the US is also reportedly progressing according to plan. The company intends to start the registration process with the US Food and Drug Administration (FDA) in the first half of 2009. (for a recent TiGenix update, click here).
Athersys received its third IND for MultiStem in less than a year with authorization to proceed on a phase I trial in the treatment of ischemic stroke.
CIRM grants were approved to 4 San Diego biotechs:
- $827,072 for Novocell to advance development of “an implantable device” to capture and retain pancreatic stem cells.
- $749,520 for a joint effort by Fluidigm of South San Francisco and StemGent, a Cambridge, MA, biotech with operations in San Diego, to develop a screening technology to help stem cell researchers “reverse engineer” skin cells into stem cells.
- $869,262 for an application by Invitrogen, now known as Life Technologies, for developing methods of modelling human neurodegenerative diseases in human embryonic stem cells.
- $906,629 for Vala Sciences to develop new and improved techniques for developing mature heart cells called cardiomyocyte cells from human embryonic stem cells.
Immunotherapy has certainly seen its share of failures but T-cell oriented therapies are not going away as they continue to attract funding by companies looking to avoid the difficulties that have sunk other efforts to commercialize immunotherapies. One such new company is Adaptimmune Ltd which recently spun out of MediGene AG to carry forward T cell receptor engineering with HIV as its initial target. The company's ex vivo approach sees a patients' own T cells harvested, those cells' receptors genetically enhanced for increased potency against certain infections or tumors in the manner of monoclonal antibody selection, and re-infused into the patient.
Osteotech authorized a stock buyback of up to $5 million.
Novocell announced it will collaborate with renowned stem cell researcher Shinya Yamanaka of Kyoto University to explore the creation of human islet cells from iPS cells. Common wisdom says Novocell has been in the lead in the race to a cell-based approach to diabetes therapy but they are not without competition. Earlier in the year giant Novo Nordisk did a deal with Cellartis and Lund University to create insulin-producing cells for diabetes. Novo Nordisk has been selling insulin since 1923 and knows the diabetes market well. Additionally, Geron announced a publication on its progress coaxing embryonic stem cells into what it calls islet-like clusters.
ERYtech Pharma announced the results of its phase II clinical trial. ERYtech Pharma, the specialty pharmaceuticals company developing red cell-based medicinal products, announces the results of its phase II clinical trial. GRASPALL 2005-1 was presented orally on the 8th December 2008 at the 50th Annual Meeting of the American Society of Hematology in San Francisco (USA). This study aimed to assess the efficacy and the safety of 3 doses of GRASPA®, a new formulation of L-asparaginase, given in frontline treatment for children and adults with
relapsed acute lymphoblastic leukemia (ALL).
In a December 3 press release and conference call ThermoGenesis announced it has reached an agreement with CEO William Osgood for his immediate resignation. This comes as a result of a clear disagreement between Osgood and Board regarding future directions of the company. A search for a new CEO will be launched. In the interim, the board has named Matthew T. Plavan, the Company’s Executive Vice President and Chief Financial Officer, as ThermoGenesis’ interim Chief Executive Officer. The company has announced its intention to reorganize, downsize, maximize, sideline its Vantus business, and dispose of the ThermoLine™ business yet this quarter.
BioLife Solutions was granted a European biopreservation patent expanding its IP related to technologies which minimize cell death during or resulting from cell preservation.
Chemokine Therapeutics Corp. (CTI.TO) filed Chapter 11 equivelant in Canada - a Proposal to Creditors under the federal Bankruptcy and Insolvency Act.
Stem Cell Therapy International Inc. announced the elimination of what it described as "major obstacles" previously blocking its merger with Histostem including the establishing of a relationship with a clinic in Mexico. The companies are apparently in the final stages of completing the merger to create AmStem International and begin creating revenue almost immediately presumably involving a new relationship with U.S. company, Cancun Stem Cell Clinic, Inc. (with its stem cell clinic in Cancun, Mexico) and Histostems cord blood banking operations.
MicroIslet Inc. is reorganizing its way through Chapter 11 with the help of new financing.
Pluristem received a $1.6M grant from the Office of Israel’s Chief Scientist. The non-dilutive funds will be designated to support the clinical trials of the Company as well as research and development activities.The grant covers the development costs of their PLX cells from March 2008 to February 2009. Furthermore, Pluristem plans to submit an application for a new grant to the OCS in March 2009 to cover the Phase I trial of PLX-PAD and pre-clinical studies of PLX-IBD, targeting Inflammatory Bowel Disease (IBD).
HCT/P establishments must complete annual registration by end of this month. Establishments that manufacture human cells, tissues, and cellular- and tissue-based products have until Dec. 31 to complete their annual registration with the Food and Drug Administration. Instructions for listing HCT/Ps have been revised this year, as have policies and procedures for submitting the form. FDA is encouraging facilities to submit the form thr obtained subject to court approval.ough electronic registration. Review the revised instructions or submit form electronically.
Interesting perspective on stem cells in biotech in BIO's year-end summary of the most important trends and stories of 2008 and looking forward to 2009.
In the spirit of passing along interesting resources, here are a couple I came across this week:
- How Stem Cell Research is Transforming Health Care. A panel discussion from the 2008 World Stem Cell Summit with panelists: Alan Trounson (California Institute for Regenerative Medicine), Lawrence S.B. Goldstein (University of California-San Diego), and John Wagner (University of Minnesota). Moderator: Timothy Kamp
- Steven Badylak @ Pop!Tech 2008. Last year regenerative medicine expert Dr. Badylak made news in the medical world when two of his patients re-grew severed fingertips in just six weeks. He spoke at this year's Pop!tech on how he and his colleagues are applying this breakthrough solution to help Iraqi war veterans re-grow tissue and body parts lost in battle.
Finally, it always disturbs me when I miss important news in the industry because it tells me there are holes in my systems. It also troubles me when I discover cell therapy companies that I here-to-fore did not know existed. Both happened to me this week. Nevertheless, better late to discovery than not at all. One of the more important bits of news I'm late to the table for (announced at the end of October) is the acquisition of "certain of the assets and the continuing business of Celltran Limited (“Celltran”) from its administrators" by York Pharma plc (AIM: YRK) for what it describes - in the very undestated British way - a "compelling valuation".
This seems an apt description when one learns that despite the fact that "approximately £15 million has been invested in the Celltran portfolio of products" to-date, York bought the company's two commercial product plus "a rich pipeline of associated products and technologies" and the ongling business for - make sure you're sitting down - "£70,000 plus the payment of royalties on future sales for a period of five years (at the greater of £100,000 (£20,000 per year) or 10% of future revenues generated by the products over such period)".
The real reason this is such a compelling valuation is that Celltran had - and now York Pharma has - two cell therapies on the market (albeit only in the UK) in Myskin™ and Cryoskin™.
But the news does not end there. York also announced simultaneous to the acquisition that they had completed an institutional placing raising £510,000 (before expenses) to provide "additional working capital for the integration, operation and further development of the assets within York Pharma’s business." York, established in 2003, describes itself as a pharmaceutical group with particular focus on dermatological products which makes the two cell therapy products fit well in their existing portfolio.
So for what its worth...that's the cell therapy industry I saw this week.
