Biotech Values

[biotech jim]

Regarding heart failure and issues leading up to that:

The big issue for drug developers is that the FDA will require an outcomes trial, and we all know or think we know what that means in terms of efficacy needed and the time and expense of such a trial or trials. With companies getting out of the cardiology arena, there will be limited players in this field.

Here is an interesting company and approach in the cardiolgy and microRNA field, in which Eric Olson is a founder. He was a founder of Myogen, a company previously bought out at a really high price by GILD. (Can anyone recall the products that GILD sells from the Myogen buyout?)

http://www.miragentherapeutics.com/

They have an interesting partnership with the private French company, Servier.

http://www.miragentherapeutics.com/105/October%2018,%202011/

Here is an abstract on some of the microRNA science:

http://www.ncbi.nlm.nih.gov/pubmed?term=Therapeutic%20Inhibition%20of%20miR-208a%20Improves%20Cardiac%20Function%20and%20Survival%20During%20Heart%20Failure

Background—Diastolic dysfunction in response to hypertrophy is a major clinical syndrome with few therapeutic options.

MicroRNAs act as negative regulators of gene expression by inhibiting translation or promoting degradation of target

mRNAs. Previously, we reported that genetic deletion of the cardiac-specific miR-208a prevents pathological cardiac

remodeling and upregulation of Myh7 in response to pressure overload. Whether this miRNA might contribute to

diastolic dysfunction or other forms of heart disease is currently unknown.

Methods and Results—Here, we show that systemic delivery of an antisense oligonucleotide induces potent and sustained

silencing of miR-208a in the heart. Therapeutic inhibition of miR-208a by subcutaneous delivery of antimiR-208a

during hypertension-induced heart failure in Dahl hypertensive rats dose-dependently prevents pathological myosin

switching and cardiac remodeling while improving cardiac function, overall health, and survival. Transcriptional

profiling indicates that antimiR-208a evokes prominent effects on cardiac gene expression; plasma analysis indicates

significant changes in circulating levels of miRNAs on antimiR-208a treatment.

Conclusions—These studies indicate the potential of oligonucleotide-based therapies for modulating cardiac miRNAs and

validate miR-208 as a potent therapeutic target for the modulation of cardiac function and remodeling during heart

disease progression.