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.