STEM ($STEM): AMD N=16 Trial gets the Green Light from the FDA

Stem Cells, Inc. (NASDAQ: STEM) gets the "green light" from the FDA to kick off a Phase I/II clinical trial of HuCNS-SC in AMD.

The Phase I/II trial will evaluate the safety and preliminary efficacy of HuCNS-SC cells as a treatment for dry AMD. While there are now therapeutic options for wet AMD there is no treatment for dry. The promise of cell therapy in this space is great. Advanced Cell Therapy (ACTC) is also pursuing this indication.

In the case of STEM, the trial will be an open-label, dose-escalation study, approx. N=16. STEM's HuCNS-SC cells will be administered by a single injection into the space beneath the retina. Patients' vision will be evaluated using conventional methods of ophthalmological assessment at predetermined intervals over a one-year period. Patients will then be followed for an additional four years in a separate observational study.

Daily Dose Conclusion: AMD is a promising area as the physical space in the eye is small and the market opportunity is large. The ability to rebuild rods & cones and restore vision is great. Several firms such as ACTC are working in the area. The key question for investors who have been patient with STEM for years and have lived through the most recent raise and reverse stock split: Is there a way back ? With Geron's (GERN) departure STEM is one of the only remaining embryonic (public) company left in the space. STEM is also pursuing the "holy grail" with a spine trial. Certainly the bet here is that STEM hits it big at some point but data is not immediate.

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Athersys ($ATHX) : GvHD Phase 1 data is encouraging...

Athersys (NASDAQ: ATHX) announced in their press release positive results from its Phase I clinical trial of MultiStem, its cell therapy product. The trial was administered to individuals undergoing allogeneic hematopoietic stem cell transplants (HSCT) for the treatment of leukemia and related conditions.

Data highlights from the study include:

• The majority of patients participating in the study received transplants from unrelated donors (19 of 36), and nearly all of the patients received peripheral blood stem cell (PBSC) transplants (34 of 36), both of which are associated with a higher risk of GvHD;
• All patients experienced successful neutrophil engraftment (median time of engraftment 15 days), and 86% of patients experienced successful platelet engraftment (median time of engraftment 16 days) which compares favorably to historical clinical experience for this patient population demonstrating a positive impact on blood and immune system recovery;
• Substantial reduction in acute GvHD incidence, relative to historical experience, at the highest single dose (11% grade II-IV GvHD, and 0% grade III-IV GvHD).

These clinical results provide the foundation for further accelerated development of MultiStem for the prevention and reduction of GvHD. Following final review of the data, and subject to input from its key scientific and clinical advisers, Athersys plans to meet with the FDA to discuss plans for the next phase of clinical development. This could include a blinded, controlled phase II/III study of MultiStem for GvHD prophylaxis and HSCT support.

Daily Dose Conclusion: Athersys is building a solid foundation to expand the pipeline in a product with Multistem.

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Athersys (ATHX): Scores Grant Money for TBI and for Formulation

Athersys, Inc. (Nasdaq:ATHX) announced $3.6 million in grant funding (actually two separate grants), one for MultiStem (allogeneic cell therapy), and the second for its clinical utility in treating traumatic brain injury (TBI). This is an area that is getting lots of attention as a result of the wars in Iraq and Afghanistan. Soldiers lucky enough to escape direct injury often suffer very severe TBI as a result of the percussion (force) related injury. The other grant goes to Athersys subsidiary in Belgium, ReGenesys BVBA, $1.2 million (€0.9 million) from the Belgium's Agency for Innovation by Science and Technology (IWT) to further develop cell therapy formulations and manufacturing capabilities.

Daily Dose Conclusion: Non-Dilutive financing is always positive and these micro-cap companies know how to make the dollars work to advance their product portfolio or in the case the potential indications for MultiStem. We believe that there is a significant role for multistem to play in TBI and even in stroke however the development costs and commercial approval in these areas is a long way off.

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ThermoGenesis ($KOOL) now in CLI ! (video)

ThermoGenesis Corp (NASDAQ: KOOL) press released an update on their Res-Q clinical evaluations:

ThermoGenesis Provides Update on Res-Q® Clinical Evaluations at Leading Stem Cell Therapy Conference

INITIAL DATA SHOW POSITIVE OUTCOMES IN TREATING CRITICAL LIMB ISCHEMIA AND LONG BONE FRACTURES

What is Res-Q ?  This is a point-of-care system designed for the preparation of cell concentrates, including stem cells, from bone marrow aspirates and whole blood for platelet rich plasma (PRP). The product is being tested in India in a trial co-sponsored by ThermoGenesis and Totipotent SC, the company's distributor for the Res-Q in India.

The trial has evaluated n=10 of n=15 planned patients with advanced CLI. Safety and AFS (limb salvage) at one year are being evaluated. Secondary endpoints such as ABI (ankle brachial index), six minute walk test, and resting pain are showing good results.

In addition a second trial is evaluating n=16 of n=20 patients with non-union or delayed union fracture of a long bone (tibia) who have undergone composite grafting with autologous bone marrow cell concentrate to evaluate the procedure's safety and effect on fracture healing. Long bone fractures represent an unmet medical need that the med tech companies have been chasing for years. So far the system has shown no intra-operative complications, and one non-device related adverse event. Of nine patients who are now out three-to-six months post-treatment, four have fractures that have united and four have experienced partial unions of the bone fracture which is quite hopeful.

Daily Dose Conclusion: Few are paying attention to KOOL as a therapeutic player in CLI or bone fractures and as a potential challenger to Cytori or even Aastrom or Pluristem in CLI. This is worth watching.

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MesoBlast ($MSB): FDA Green Lights PII Trial for Type 2 Diabetes

MesoBlast (ASX: MSB) press released FDA approval to move to a PII trial today. Recall that MSB's product is "allogeneic" (other people's cells) and as such is "off-the-shelf" ready. Similar to Athersys and PluriStem approaches. Diabetes is a large market and is often related to an auto-immune condition that results in damage to the insulin producing cells (islets of the pancreas).

The MesoBlast trial will be a randomized, placebo-controlled, 3 month trial that will evaluate a single IV injection of one of three (progressively higher doses of cells) in N=60 (Type II diabetes patients) who show elevated blood glucose levels. Animal data in this indication showed hints of activity. MesoBlast has demonstrated safety with their product in several prior trials.

Daily Dose Conclusion: MesoBlast recognizes that their cell therapy product may have multiple uses and multiple indications. MesoBlast had a BIG WIN when Cephalon invested in their therapy (since acquired by Teva). With that said the market cap of MesoBlast peaked back in November and has come down sharply (>40%) as investors have struggled with the high valuation. The cardiology data presented at AHA seemed to mark the peak. Valuations for Mesoblast versus other Allogeneic companies in the space such as Athersys and PluriStem are tough to understand.

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Prima BioMed ($PRR) Granted Japanese Patent for Cripto-1 Antibody; $PRR

Prima BioMed (ASX: PRR) is an Australian health care  company focused on technologies in the fields of cancer immunotherapy and immunology. Prima’s lead product is the CVac (tm) ovarian cancer therapy treatment. It has completed two successful clinical trials and is progressing toward eventual commercialisation in the United States, Australia, Europe, and globally. The Company’s broader, long term goal is to develop commercial cancer treatment technologies and programs for global markets.

One of the company's three programs in place is focused on the identification and development of a monoclonal antibody to cripto-1. Monoclonal antibodies have been a major success in recent improvements of cancer treatment. Cripto-1 is a component of the endothelial growth factor complex, and neutralizing this important protein will enable Prima to target multiple tumour types.

The company announced that it "has received Notification of Grant by the Japanese Patent Office for its antibody to cancer antigen Cripto-1. Patent number 4854912 is titled, Antibodies against Cancer and expires in March 2022. The patent has also been granted in Australia, New Zealand, China, South Korea and the USA."

The granted patent claims "protect the use of an antibody directed to the cancer antigen Cripto-1. Cripto -1 is a protein found in high levels on the surface of many different kinds of cancer cells and is also found to circulate in the blood stream of cancer patients. The antibody works by binding to the Cripto-1 molecule and interfering with cell signalling, resulting in the death of the cancer cell. The antibody may additionally be coupled to cytotoxic drugs to deliver a more lethal combination of drug and cell signal to cancer cells."

Read the full release on PrimaBioMed.com.

ThermoGenesis Corp (KOOL) - ReOrganizing ?

ThermoGenesis Corp (NASDAQ: KOOL) announced a management transition in conjunction with tactical realignment of company in this press release.

Thermogenesis is an automation/tool's supplier in and around the world of cell therapy. The company has had their struggles with the business model and a run towards profitability which also seems to be a year away. News today that Matthew Plavan (currently CFO / EVP, Business Development), will become CEO and a board member as well as the CFO is tough to read. According to the press release:

• Plavan will replace J. Melville Engle, (who has retired from his position as Chairman and CEO).
• The company will cut eight additional positions and provide additional details on its new operating structure and objectives during its Q2 fiscal 2012 conference call on Thursday, 9-Feb.
• KOOL expects to record one-time expenses of approximately $500,000 related to the reorganization announced in Q3 of fiscal 2012 BUT the restructured operations should result in an annualized expense reduction of approximately $2M (according to KOOL.

Daily Dose Conclusion: No Rush to jump into KOOL on a reorg news but this is a great company, strong technology that at some point could be acquired by a bigger fish.

SANUWAVE Health (SNWV): Pulsed Energy and Stem Cells ? Stay Tuned !

Last week at the Cell Therapy Cardiology Conference held at Columbia Presbyterian Hospital we heard KOL's (Key Opinion Leaders) from just about every stem cell company at the meeting. Several presentations held great interest for all, one in particular from Dr Zeiher of t2cure was quite interesting. t2cure (German - private) has been working on bone marrow derived cell therapy product for AMI. According to Dr Zeiher (founder) pre-conditioning cardiac ischemic patients with sound wave energy (much like the probes that are used to break up kidney stones). The company believes that they can pre-condition ischemic hearts, active a homing signal (stromal derived factor-1 SDF-1) that will allow the active cells in marrow (CD34+ / CXCR4+) cells to lock in on the ischemic area and create new blood vessel formation.

Daily Dose Conclusion: Sanuwave (OTCBB: SNWV) has demonstrated that their device does enhance healing and probably through the mechanism that Dr Zeiher was presenting last week. Sanuwave missed their primary endpoint (100% wound closure in diabetic foot ulcers - DFU) but they really only just missed it. Wounds that had not healed without their therapy healed 99%. The last 1% is almost impossible to get perfect closure, and as a result, the FDA has requested they do another trial. While we are not sure what the action plan at Sanuwave will be, we do see this technology as important to the stem cell space.