AtheroGenics has announced that its lead drug candidate, AGI-1067, failed a Phase III clinical trial because it did not reduce the length of time before patients suffered one of six cardiovascular events.
These included the need for coronary revascularization and admission to hospital for unstable angina.
However, there is hope for the US biotech after the drug did reduce the number of heart attacks, strokes and led to an improvement in several key diabetes parameters, despite these events being seen as harder to prevent.
"While we are obviously disappointed with the primary endpoint results announced today, we believe that at this point in time, based on the results we have reviewed, we are encouraged and optimistic that further development of 1067 is warranted in a number of important therapeutic areas," said Dr Russell Medford, CEO of AtheroGenics.
If approved the drug would be classed as a new chemical entity (NME) by the US Food and Drug Administration (FDA) as it works via a novel mechanism of action.
The drug was developed to reduce inflammation through blocking a protein called VCAM-1.
Through binding to VCAM-1, the small molecule drug prevents the protein from helping white blood cells stick to artery walls.
Normally these white blood cells are thought to attack the endothelial cells that line the wall of the artery and the resulting inflammation leads to plaques being formed, which in turn causes atherosclerosis - a hardening of the arteries.
However, several other drugs affect VCAM-1 levels and their development could be influenced by the results for AGI-1067.
For example, scientists at the University of Tokyo are developing a drug (K-7174), which prevents the protein being produced through binding to its gene promoter region.
The drug has also been shown to be selective and not interfere with the production of other inflammatory proteins, such as NFkappaB. Medford went on to say the company are working with the FDA on the next steps that could enable AGI-1067 to be approved.
The company's partner on the drug, AstraZeneca, has 45 days to decide whether to continue with the collaboration.
AstraZeneca originally pledged up to $1bn (€752m) to help develop the drug, although this figure did depend on the drug's success.
Medford went on to point out that the time limit does not begin until a full analysis of the trial has been completed.
He also added that AtheroGenics has enough cash reserves to pursue development of the drug on its own if necessary.