ARM Panel shines light on the Cell Therapy Cardiovascular Space

This morning in SanFransico the ARM panel on cardiovascular medicine is taking place. NeoStem (Amorcyte), MesoBlast (MSB), Athersys (ATHX) and Cytori (CYTX) are presenting their cell therapy approaches. We will follow up with comments from this panel and the panels that follow throughout the day. In other news NeoStem announced the expansion of their IP for AMR-001 (their CD34+ cell therapy product) beyond STEMI (heart attacks) and to all ischemic (vascular insufficiency) diseases. We looked at the patent and we see this as a landmark expansion in the space. Immunocelluar (IMUC) also had news today, raising capital (aprox $9.4 mln) which will be used to fund the development of their dendritic approach to brain cancer (glioblastoma). We note that IMUC is working with NeoStem's CMO subsidiary, Progenitor Cell Therapy (PCT).

The TurnAround in Dendrion ? DNDN Presents at JP Morgan in SFO

Last week Dendreon NASDAQ: DNDN reported stronger than expected sales for Provenge sales at $82 Million generally above "street" consensus expectations. The company has a strong cash balance (approx. $600M) to fund the Provenge launch through to profitability. The stock rallied sharply starting the week at $8 and closing north of $12. This sets the stage for continued expansion for Dendrion and is welcome news for earlier stage cancer immune based therapies in development.

This week the JP Morgan Healthcare Conference begins in San Fransico, Monday through Thursday. Healthcare companies from all over the world converge for a week in meetings with sell side analysts, bankers and institutional portfolio managers.

We have been highlighting the Alliance for Regenerative Medicine (ARM) efforts as an industry group to highlight the progress that cell based companies have made over the past decade. To this end, ARM has organized a two day mini-conference (we have previously posted the schedule). What's special about the ARM event is that the companies will all present in a panels style format.

Industry leaders like Dendrion (presenting on a panel Wed) will be alongside other earlier stage companies in their market niche such as Immunocellular (IMUC), Coronado (CNDO), PrimaBioMed (PRR.AX) as well as other players in the space. We believe this will be a great venue for investors to learn not only about where Dendrion may be going as a stock, but what's next in the cancer-immunotherapy space.

We will be present throughout the day and blogging on the various panels as we go through the motions of comparing and contrasting the various approaches of the companies. For example we are keenly focused to listen to the cardiology panel where industry leader MesoBlast (MSB) will present alongside NeoStem (Amorcyte-NBS), Athersys (ATHX) and Cytori (CYTX).  Baxter (BAX) will not be present for the panel but investors should take note of their progress (P3 trial in cardiac ischemia).

We believe the next major breakthroughs on the regen side will likely be found in the cardiology space.

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.