Immunocellular ($IMUC) - Moving forward in Brain Cancer

IMUC is developing ICT-107, an autologous, dendritic cell-based vaccine (like Dendrion's Provenge) that works by activating a patient's immune system against specific tumor-associated antigens. This is accomplished by extracting dendritic cells from a patient, loading them with the antigens, and reintroducing them to the patient's body to trigger an immune response. It is still early days, but IMUC is targeting glioblastoma multiforme (aggressive brain cancer) with this product. Data from Phase 1 trials has been encouraging and the company is embarking on a P2 program. The company is also developing ICT-140: Dendritic cell-based vaccine for the treatment of ovarian cancer. This product will target multiple antigens present on cancer stem cells and daughter cells. Interesting because we have discussed Prima BioMed's dendritic approach in ovarian cancer too.

This is not new news, but its worthy of mention, according to a blurb we saw in Bio Century, IMUC announced in early December the company’s plans to raise capital in a follow-on offering with a goal to raise up to $13.8 million in cash (10 million at $1.40) with 1:1 share – warrant coverage.

Fate Therapeutics - William Rastetter becomes Chairmen & Interim CEO

William Rastetter PhD is best known for his time at Idec Pharmaceuticals (can you say rituxan). He is considered a heavyweight in the biotechnology world so his willingness to take on this role at Fate Therapeutics is not only positive for Fate but for the industry overall. Fate Therapeutics is working on ProHema (umbilical cord blood transplants (UCB), with data recently presented at ASH.

Stem Cells Inc. (STEM): Prices $10M secondary at $1.25

In a surprise offering Stem Cells Inc. (NASDAQ: STEM) announced the pricing of 8 million share offering with matching warrants for gross proceeds of $10M, at the bottom of what has been a declining valuation. Earlier in the week we discussed the perfect storm, the macro-economic outlook coupled with declining balance sheets has hurt biotechnology companies that have "miles to go" before they can reach commercialization. Dendreon's implosion and Geron's departure did not help. As a result valuations have declined for everyone in the cell therapy space. Raising capital at the bottom hurts, but it is the nature of biotechnology and the rule here is dilution versus extinction.

It is surprising to us that the company announced earlier in the week news that the first cohort of the Phase I/II trial in chronic spinal cord injury has been treated (successfully transplanted with the STEM's proprietary HuCNS-SC® neural stem cells).

This trial has a unique design, in which patients with progressively decreasing severity of injury will be treated in three sequential cohorts. The first cohort of patients all have spinal cord injury classified as AIS A, the most severe level identified by the American Spinal Injury Association Impairment Scale (AIS).

We have seen Dr. Stephen Huhn MD, FACS, FAAP, (Vice President and Head of the CNS Program at StemCells) present in the past. He is considered a KOL (Key Opinion Leader) in the field and is highly respected. He is quoted:

"Having completed dosing of the AIS A cohort, screening for AIS B patients, who have a less severe, incomplete type of spinal cord injury, can now begin. Of course, our first priority is to assess safety in each patient, but we will also be evaluating trial patients for changes in sensation, motor and bowel/bladder function."

It’s still early days for STEM and impossible to know if these cells will translate into any efficacy but the good news is that this raise does give the company some runway to see a signal from this trial. It’s a longshot for sure and there are other companies in the space with more mature trials, defined mechanisms of action, dose responses defined, large target markets, and great efficacy signals established in P1 and 2 trials. Look at the developments in cardiology (AMI / CHF) - Mesoblast, Cytori, NeoStem, where we believe proof of concept is coming, but the holy grail of stem cell therapy is to be able to show a signal, anything, in paralysis. STEM now will carry that banner.

Bio Smart Brief : Survey Picks Stem Cells

Bio SmartBrief circulated an Expert Views and Insights piece yesterday. We highlight one of the posted survey sections:

Poll: What field is most likely to yield the biggest breakthrough in the coming year?

23%		Stem-cell therapies
14%		Specific disease therapies -- HIV, cancer
14%		Vaccines
12%		Someplace unexpected
10%		Genomic discoveries
8%		Advanced biofuels
8%		Agricultural biotech
7%		Gene therapy
4%		Synthetic biology

Conference on Cell Therapy for Cardiovascular Disease (January 25-27) NYC - Baxter & More...

We picked up on an advertisement from the Cardiovascular Research Foundation for the seventh International Conference on Cell Therapy for Cardiovascular Disease. January 25-27, 2012 in New York. What struck is the lineup of the companies and panelists and the implications for the cardiovascular companies in the space, such as Baxter, Cytori, MesoBlast, NeoStem.

What caught our eye is the panel on the second day, "Acute Injury: STEMI and NSTEMI". The panel will be moderated by some very well-known KOL's such as Henricus J. Duckers, Gregg W. Stone, Andreas M. Zeiher. Dr Zeiher is one of the founders of a German private company also working on a cell based therapy (cells that follow an ischemic gradient) for AMI. Cytori will talk about the PRECISE trial (Adipose (from fat) stem cells for chronic ischemia. Dr. Doug Losorodo (who is not the principal investigator in the Baxter phase III trial) which uses CD 34+ cells (just like NeoStem (NBS) Amorcyte trial) will also present on the following panel which will review CLI (Critical limb ischemia) and CMI (Chronic Myocardial Ischemia). Even Dr. Robert Bolli who was recently features on CBS talking about his myocytes that repair dead heart muscle will be a panelist.

Our take away message: When we see Baxter and other prominent researchers now talking in these forums , presenting data and defined mechanisms of action, it raises our confidence level that proof of concept from trials is not far behind. Watch the work that NeoStem (NBS) is doing in AMI as a value play versus Baxter (BAX) with a very similar therapeutic approach. Doug Losordo (PI Baxter) has been working in the space for 20 years and adds a measure of credibility as do the entire panel members.

Link to the Conference web site: http://celltherapy.crf.org/home.html

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Day 2 Panel Agenda

=============================

Acute Injury: STEMI and NSTEMI

8:30 am - 9:55 am

Moderators: Henricus J. Duckers, Gregg W. Stone, Andreas M. Zeiher

8:30 am

Trial I. Long-term FU of Patients with an AMI Treated with Adipose-Derived Stem Cells (APOLLO)

Henricus J. Duckers

8:40 am

Trial II. LATE-TIME and Others

Stephen G. Ellis, Timothy D. Henry

8:50 am

Trial III. MAPC Results and Directions

Marc S. Penn, Warren Sherman

9:00 am

Trial IV. TBA

Raj Makkar

9:10 am

EPTS

Henricus J. Duckers, Marc S. Penn

9:25 am

Discussion

Break

9:55 am - 10:25 am

CLI/CMI

10:25 am - 12:00 pm

Moderators: Francisco Fernandez-Aviles, Amit Patel

10:25 am

Trial I. CD34+ Cells for Refractory Angina

Douglas W. Losordo

10:35 am

Trial II. Adipose-Derived Cells for Chronic Ischemia: The PRECISE Trial

Francisco Fernandez-Aviles

10:45 am

Trial III. Update on Standardization of CD133 Intramyocardial BMSC

Gustav Steinhoff

10:55 am

Trial IV. BMAC for CLI: Status of a Phase 3 Trial

Thomas F. O'Donnell

11:05 am

Trial V. Placenta-Derived Cells for CLI

Christof Stamm

11:15 am

Abstract Presentation

11:20 am

EPTS

Timothy D. Henry, Amit Patel

11:35 am

Discussion

Lunch

12:00 pm - 1:00 pm

Heart Failure and Chronic Disease

1:00 pm - 2:30 pm

Les Miller

1:00 pm

Trial I. SCIPIO

Roberto Bolli

1:10 pm

Trial II. Cell Wave

Andreas M. Zeiher

1:20 pm

Trial III. SDF-1 and SDF-2

Marc S. Penn

1:30 pm

Trial IV. Cardiocyte-Driven Bone-Marrow Precursors

Jozef Bartunek

1:40 pm

Trial V. MPC's for Ischemic and Non-Ischemic Heart Failure

Emerson C. Perin

1:50 pm

EPTS

Jozef Bartunek, Randall J. Lee, Amit Patel

2:05 pm

Discussion

Celladon is focused in Heart Failure...gets Fast Track Designation (private)

Cell Therapy fan's know that on the Regenerative Medicine front the next major breakthrough may very well be in cardiovascular medicine. Several companies are in trials now with a wide range of cell therapies to stabilize failing hearts. What caught our eye today is news that private biotech company Celladon received fast track designation for Mydicar.

Celladon's MYDICAR® is a genetically targeted enzyme replacement therapy that is designed to restore functional levels of "SERCA2a", (a regulator of calcium cycling and contractility). In other words, this enzyme is required for the heart to beat properly and in patients whose heart is failing leading the enzyme levels to decline. MYDICAR® is a gene that is delivered using a recombinant adeno-associated virus (AAV) as the vector. This is really high tech gene therapy at a cellular level.

So what does this have to do with regenerative cell therapy ?

The phase 2 trial results (previously announced) showed the therapy met its primary safety and efficacy endpoints at 6 months for high dose MYDICAR® versus placebo. Additionally, 12 months after receiving a single infusion of MYDICAR®, patients treated with the highest dose versus placebo had an 88% risk reduction (Hazard Ratio = 0.12, P=0.003) of major cardiovascular events such as death, or a need for a left ventricular assist device (LVAD), cardiac transplant, or episodes of worsening heart failure and the related heart failure hospitalizations.

So now we are starting to see cardiac trials with therapies that are reporting a preservation of heart function and risk reduction as the therapeutic goals. We believe that in the cell therapy space, we will see a host of therapies developed along these same lines. We would take note on the autologous side of Baxter (P3) trial, NeoStem (NBS) (P2) trial, Aastrom (P2) and on the allogeneic side, MesoBlast (MSB), Cytori (CYTX), Athersys (ATHX), and others.

Critical Limb Ischemia (CLI) gets a lot of attention with Aastrom (ASTM), PluriStem (PSTI) and Aldagen (private) all talking about the potential but investors have not been convinced. We would watch closely the developments on the cardiac side as the unmet medical need here remains large. 

Why? The historical data sets and trial designs are well understood, so the feasibility of a successful trial based on what's known about the space is positive. News like Celladon, and a close examination of the trial goals bode well for the space.

Tengion (TNGN) : NeoUrinary Conduit

Tengion had a press release earlier today, announcing that they will have several presentations on the company's Neo-Urinary Conduit™ (NUC) and Neo-Kidney Augment™ (NKA) programs at the TERMIS North America Annual Conference now being held (December 11-14), in Houston, TX. Reading the press release tells us that the data set is building for both the NUC and NKA programs. We believe that the market size on the kidney program is off the charts, but acknowledge that the program is still pre-clinical so it’s not a driver for the stock yet.

What we find striking here is the fact that Tengion was a rising star. IPO'd by two of the best healthcare Wall Street firms and since that time, the stock has plunged, now trading at a little over a $10 million market cap with some trading liquidity. According to the September SEC filing, the Company had $9 mln in cash and $11 mln in short term investments versus $1.9 mln ST debt and $3.3 LT debt.

We have seen the NUC device and the technology is impressive. We see a company like Tengion, and fast followers in the space like Organovo as breakthrough technology plays. Perhaps we will see interest from Medical technology companies who are more familiar with the intense surgical procedures and specialized markets for devices that take interest in a product like NUC.

Our View: We would not give up on Tengion here.

See all TNGN articles @ Daily Dose Equities.

Neo-Urinary Conduit: Oral Presentation

• Genetic Stability of Autologous Human Smooth Muscle Cells (http://www.tengion.com/news/documents/Justewicz-SMC-Stability-Tengion-TERMIS-NA-Houston-14Dec11.pdf)
• Characterization of Chemotherapy and/or Radiation Treated Human Smooth Muscle Cells for Use in Regenerative Medicine (http://www.tengion.com/news/documents/Justewicz-Cancer-Secretome-Tengion-TERMIS-NA-13Dec11.pdf)

Neo-Kidney Augment : Oral Presentations

• Selected Regenerative Renal Cells combined with Bioresorbable Natural Biomaterials Catalyze in vivo Kidney Tissue Regeneration (http://www.tengion.com/news/documents/2011-Jain-TERMIS-NA-Presentation.pdf)

• Multivariate Analysis of Preclinical Data from the Tengion Neo-Kidney Augment (http://www.tengion.com/news/documents/Spencer-TERMIS-Presentation_121411_rev2.pdf)

Cell Therapy - Oncothyreon ($ONTY) presents data, Prima BioMed update

A few news events over the last 24 hours caught our attention and in effort to connect the dots we are highlighting them in the context of what impact it may have on the space. Oncothyreon ($ONTY) presented data (actually their partner Merck KGaA) at the American Society of Hematology on Stimuvax.

Stimuvax is a cancer vaccine that targets the MUC-1 antigen (same one that Prima's CVAC targets). The data presented was from the phase 2 trial in Multiple Myeloma (MM). Two phase 3 trials are now ongoing:

• Lung cancer "START" trial, and
• "INSPIRE" (Asian patients).
  

The Phase 2 data presented in Multiple Myeloma looks early but encouraging. The next big data point that investors are watching is the interim look (the second one) for the P3 trial, which should happen in Q3-2012.

What investors may not realize is that PrimaBioMed (ASX: PRR) is also targeting the MUC-1 antigen but using sensitized dendritic cells (just as Dendreon has done successfully with Provenge). PrimaBioMed CEO Martin Rogers has been working on listing this Aussie stock on a US exchange. We think watching Oncothyreon and its impact on PrimaBioMed will be an important connection for investors to watch. See Prima BioMed @ ProActive.