Researchers at the Karolinska Institute in Sweden have reported the results of an early-stage clinical trial of a new oral drug, AC01, for patients with chronic heart failure. The findings were published in The Lancet.

Heart failure with reduced pumping function means the heart is unable to pump blood efficiently throughout the body. Although several treatment options are currently available, many patients continue to experience disease progression over time. Moreover, medications that increase the strength of heart contractions are often associated with serious side effects, including cardiac arrhythmias and changes in blood pressure.

The investigational drug AC01 targets the ghrelin receptor, which is best known for its role in regulating metabolism and growth hormone secretion. This receptor is also expressed in heart muscle, allowing researchers to improve cardiac contractility through an alternative biological pathway.

The randomized, placebo-controlled phase 1b/2a clinical trial enrolled 58 patients with stable chronic heart failure. Participants received different doses of AC01 or a placebo for either seven or 28 days. The primary objective of the study was to evaluate the drug’s safety and tolerability.

The results showed that AC01 was well tolerated. No serious treatment-related adverse events were reported, and the drug did not negatively affect heart rhythm or blood pressure.

Additional analyses suggested potential improvements in cardiac function, including increases in stroke volume and cardiac output. However, the researchers emphasize that this was an early-stage study involving a relatively small number of participants.

According to Professor Lars Lund of the Karolinska Institute and Karolinska University Hospital, the findings support conducting larger and longer clinical trials to determine the drug’s clinical efficacy.

The study was conducted in collaboration with medical centers across several European countries and was funded by the pharmaceutical company AnaCardio AB.