German biotech company Cardior Pharmaceuticals’ candidate heart failure drug has the potential to stop the condition in its tracks, according to early studies in animal models.
At present, treatments for heart failure, including ACE inhibitors, beta blockers, and diuretics, target the symptoms of the disease, such as high blood pressure, rather than the actual cause. There have been no new curative or mechanistic drugs for heart disease in the last 20 years, so demand is high.
Cardior’s drug “for the first time directly modulates causal pathological pathways in the heart and has the potential to be a curative therapy,” Thomas Thum, CSO and founder of Cardior, told me.
Cardior’s CEO and co-founder Claudia Ulbrich added: “Our drug is an inhibitor acting on a certain microRNA that controls three distinct pathways responsible for the remodeling of the heart in heart failure. Therefore, the mode of action completely differs from all current drugs on the market or under development.”
The noncoding microRNA that Ulbrich described is known as miR-132, which is overexpressed in a number of pathologic heart conditions in both humans and animals. Cardior’s lead drug blocks the action of this microRNA.
The research, published in Nature Communications, shows that the drug reduced the levels of miR-132 in the cardiac tissue of mice with heart failure, which resulted in an improvement in cardiac function. This was partly due to the drug reversing the detrimental effect that elevated miR-132 had on the heart’s ability to contract correctly.
These findings led the researchers to investigate the compound in pigs with heart failure, a larger clinical model that better mimics the situation of people who have developed heart failure following a heart attack. This model showed that two doses of the drug improved cardiac function significantly better than the placebo. There was also no evidence of any drug-related side-effects.
Cardior’s drug is now being tested in a phase 1b trial among people with stable ischemic heart failure, with the results expected in late 2020.
Thum believes that, as the mechanism of action is similar across various subtypes of heart failure, the drug has a large market potential. He explained that up to 50% of heart failure is due to a form of the condition called ‘heart failure with preserved ejection fraction’ — where the left ventricle is able to pump normally but fills up with less blood than healthy ventricles — for which there are currently no approved drugs.
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