Cell-based Cancer Immunotherapies. Some metrics..

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Whatever one makes of Dendreon's challenges in bringing Provenge to market and then its ups and downs in the market, the whole affair has brought a much bigger spotlight to cancer immunotherapies and cell-based immunotherapies in particular.

This is true on all fronts.  Cancer immunotherapy conferences are popping up everywhere.  A growing number of of analysts are now covering a growing number of companies in the space with coverage ranging from bearishly critical to ebullient bullishness. Some venture capital firms are now loosening their purse strings for immunotherapy plays and both pharma venture funds and business development departments are now spending an increasing amount of time actively monitoring and exploring potential plays in the sector.

One of the best annual industry summaries of what is happening in the sector is sponsored by MD Becker Partners through its annual Cancer Immunotherapy: A Long Awaited Reality conference held each in New York, this year on October 4 and select video replays it hosts on its YouTube channel.

Some Segment Metrics

As part of our ongoing industry intelligence and consulting services we actively track the activity and progress of industry-sponsored clinical trials of all cell therapies in addition to the products already on the market.  Here's how our data stacks up regarding the cell-based immunotherapies segment of the sector:

Commercial:

• Dendreon's Provenge
• Autologous immunotherapy for prostate cancer (1 monthly dose for 3 months)
• Efficacy: prolongs survival
• Markets: only the United States (approved April 2010)
• Next markets: submitted the marketing authorisation application to the EMA (European Medicines Agency) in early 2012 and hopes to introduce Provenge in the European market in 2013
• 2011 Revenue $290M
• Projected 2012 Revenue: ~$380M

Phase III or II/III:

• Argos Therapeutics' AGS-003
• phase III trial not yet recruiting
• Indication: advanced renal cell carcinoma
• Estimated enrollment: 450
• Estimated primary completion: June 2014 

• Cell Medica's Adoptive Cellular Therapy (ACT) 
• phase III trial actively recruiting
• Indication: cytomegalovirus infection following allogeneic stem cell transplantion
• Estimated enrollment: 90
• Estimated primary completion: June 2013

• JW Creagene's CreaVax-RCC 
• phase III trial actively recruiting
• Indication: metastatic renal cell carcinoma
• Estimated enrollment: 54
• Estimated primary completion: unknown

• Kiadis Pharma's alloreactive T-cells (ATIR)
• phase II/III trial currently suspended
• Indication: infections and relapse assoc’d w/ SCT for leukemia and other hematologic malignancies
• Estimated enrollment: 70
• Estimated primary completion: TBD

• MolMed's TK
• phase III trial actively recruiting
• Indication: acute leukemia
• Estimated enrollment: 170
• Estimated primary completion: January 2014

• Newlink Genetics' HyperAcute®-Pancreas (algenpantucel-L)
• phase III trial actively recruiting
• Indication: pancreatic cancer
• Estimated enrollment: 722
• Estimated primary completion: January 2014

• NovaRx's Lucanix
• phase III trial actively recruiting
• Indication: non-small cell lung cancer
• Estimated enrollment: 506
• Estimated primary completion: June 2012

• PrimaBioMed's Cvac
• phase II/III trial actively recruiting
• Indication: epithelial ovarian cancer
• Estimated enrollment: 1,000
• Estimated primary completion: March 2015

Phase II or I/II

• 50 industry-sponsored clinical trials of cell-based immunotherapies actively recruiting, active no longer recruiting, active not yet recruiting, or anticipated to commence yet in 2012
• ~10 of these are expected to have readouts yet this year
• Trial sites in US, Canada, UK, continental Europe, Israel, South Korea, India, Australia
• Expected enrollment of 3,500+

Investment:

The following are notable cash infusions into the sector for 2012 to-date:

• Bellicum Pharmaceuticals.  $20M series B. 
• CellMedica. $15M grant from CPRIT in Texas.
• Argos Therapeutcs. $25M Series D.
• Northwest Bio.  $5.5M grant from German gov't Saxony Development Bank

Hope this is useful.

--

This post has been brought to you by your friends at CTG.  All cell therapy. All the time. :)  

-- Lee @celltherapy

p.s.  As always we welcome your feedback, comments, and corrections.  

Cell Therapy Industry HiLites 2008-12-12

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.